Portal de investigación
- Inicio
- Proyectos y Estudios
- ESTUDIO DE RETIRADA ALEATORIZADO EN FASE II, CONTROLADO CON PLACEBO Y DOBLE CIEGO CON RECLUTAMIENTO ENRIQUECIDO PARA EVALUAR LA EFICACIA Y LA SEGURIDAD DE BIIB074 EN EL TRATAMIENTO DEL DOLOR SUFRIDO POR SUJETOS CON NEUROPATÍA DE FIBRAS PEQUEÑAS CONFIRMADA E IDIOPÁTICA O ASOCIADA A DIABETES MELLITUS.
ESTUDIO DE RETIRADA ALEATORIZADO EN FASE II, CONTROLADO CON PLACEBO Y DOBLE CIEGO CON RECLUTAMIENTO ENRIQUECIDO PARA EVALUAR LA EFICACIA Y LA SEGURIDAD DE BIIB074 EN EL TRATAMIENTO DEL DOLOR SUFRIDO POR SUJETOS CON NEUROPATÍA DE FIBRAS PEQUEÑAS CONFIRMADA E IDIOPÁTICA O ASOCIADA A DIABETES MELLITUS.
Datos básicos
- Protocolo:
- 802NP206
- EUDRACT:
- 2017-000991-27
- NCT:
- Centro:
- HOSPITAL UNIVERSITARI I POLITÈCNIC LA FE
- Año de incio:
- 2018
- Año de finalización:
- 2020
Documentos
- No hay documentos
Participantes
Financiadores - Promotores
Resultados del Ensayo Clínico
[Translated article] Cybersecurity: a priority for pharmacy services in the age of artificial intelligence
Marin, Cayetano M. Hernandez, Monte-Boquet, Emilio, Andres, Jose Luis Poveda
Editorial Material. 10.1016/j.farma.2024.08.009. 2024
[Translated article] Good humanisation practices in the healthcare of patients with rare diseases in Pharmacy Services
Company Albir MJ, Andrés JLP, Solsona MDE
Article. 10.1016/j.farma.2023.10.004. 2024
[Translated article] New challenges in advanced therapies.
Megías-Vericat JE; (...); Poveda Andrés JL
Article. 10.1016/j.farma.2024.05.001. 2024
[Translated article] Pharmacokinetic-guided switching from standard half-life factor VIII to extended half-life pegylated factor VIII in haemophilia A therapy in clinical practice.
Chovi-Trull, Maria; (...); Andres, Jose Luis Poveda
Article. 10.1016/j.farma.2024.12.007. 2025
[Translated article] Real-world effectiveness and safety of nivolumab in patients with relapsed or refractory classical Hodgkin lymphoma.
Fernandez, Laura Lorente; (...); Andres, Jose Luis Poveda
Article. 10.1016/j.farma.2024.09.006. 2024
2019 SEFH National Survey: service portfolio, care activities, education and research in Spain's hospital pharmacy departments.
Pérez-Encinas M; (...); Calleja-Hernández MÁ
Article. 10.7399/fh.11611. 2021
A description of variant transthyretin amyloidosis (ATTRv) stage 1 patients and asymptomatic carriers in Spain: the EMPATIa study
Davila, Lucia Galan; (...); Tarilonte, Patricia
Article. 10.1186/s13023-024-03304-9. 2024
A Multicenter, Double-Blind, Placebo-Controlled, Pivotal Phase III Study (PLEO-CMT) of a Fixed Combination of Baclofen, Naltrexone and Sorbitol (PXT3003) for Charcot-Marie-Tooth Disease Type 1A (CMT1A)
Attarian, S; (...); Goedkoop, R
Meeting Abstract. 2018
A multicriteria decision analysis (MCDA) applied to three long-term prophylactic treatments for hereditary angioedema in Spain
Zozaya, Neboa; (...); Hidalgo-Vega, Alvaro
Article. 10.33393/grhta.2022.2333. 2022
A multi-criteria decision analysis on the value of nintedanib for interstitial lung diseases.
Zozaya, Neboa; (...); Verde, Luis
Article. 10.1017/S0266462322000459. 2022
A Multiple Stakeholder Multicriteria Decision Analysis in Diabetic Macular Edema Management: The MULTIDEX-EMD Study.
de Andrés-Nogales F; (...); Martínez-Olmos J
Article. 10.1007/s41669-020-00201-2. 2020
A MULTI-STAKEHOLDER MULTICRITERIA DECISION ANALYSIS IN DIABETIC MACULAR EDEMA. MULTIDEX-EMD STUDY.
Casado, MA; (...); Grp MULTIDEX-EMD
Meeting Abstract. 10.1016/j.jval.2018.09.2545. 2018
A new definition and refocus of pharmaceutical care: the Barbate Document
Morillo-Verdugo R; (...); Poveda-Andrés JL
Article. 10.7399/fh.11389. 2020
A novel TRMT5 mutation causes a complex inherited neuropathy syndrome: the role of nerve pathology in defining a demyelinating neuropathy.
Argente-Escrig, Herminia; (...); Sevilla, Teresa
Article. 10.1111/nan.12817. 2022
A Roma founder BIN1 mutation causes a novel phenotype of centronuclear myopathy with rigid spine
Cabrera-Serrano, M; (...); Paradas, C
Article. 10.1212/WNL.0000000000005862. 2018
A study of the phenotypic variability and disease progression in Laing myopathy through the evaluation of muscle imaging.
Muelas N; (...); Vilchez JJ
Article. 10.1111/ene.14630. 2021
A very mild phenotype of Charcot-Marie-Tooth disease type 4H caused by two novel mutations in FGD4
Argente-Escrig, H; (...); Sevilla, T
Article. 10.1016/j.jns.2019.05.015. 2019
A very mild phenotype of Charcot-Marie-Tooth disease type 4H caused by two novel mutations in FGD4
Argente-Escrig, H.; (...); Sevilla, T.
Meeting Abstract. 2019
Adherence and adverse events of intraperitoneal chemotherapy in optimally debulked ovarian cancer patients: Real-life study.
Ballesta-López O; (...); Poveda-Andrés JL
Article. 10.1177/1078155220915953. 2020
Analysing criteria for price and reimbursement of orphan drugs in Spain.
Badia, X; (...); Tort, M
Article. 10.7399/fh.11147. 2019
Analysis of chemical contamination by hazardous drugs with BD HD Check(R) system in a tertiary hospital
Valero García S; (...); Poveda Andrés JL
Article. 10.1177/10781552211038518. 2021
Analysis of Juvenile Onset Pompe Disease patients included in the Spanish Pompe Registry
Martinez Marin, R.; (...); Diaz Manera, J.
Meeting Abstract. 2022
Analysis of Juvenile onset Pompe disease patients included in the Spanish Pompe Registry
Martinez Marin, R.; (...); Diaz Manera, J.
Meeting Abstract. 10.1016/j.nmd.2022.07.144. 2022
Analysis of muscle magnetic resonance imaging of a large cohort of patient with VCP-mediated disease reveals characteristic features useful for diagnosis.
Esteller D; (...); Díaz-Manera J
Article. 10.1007/s00415-023-11862-4. 2023
Analysis of the diagnostic pathway and delay in patients with amyotrophic lateral sclerosis in the Valencian Community.
Vázquez-Costa JF; (...); Sevilla-Mantecón T
Article. 10.1016/j.nrl.2018.03.026. 2018
Analysis of the diagnostic pathway and delay in patients with amyotrophic lateral sclerosis in the Valencian Community.
Vazquez-Costa, J F; (...); Sevilla-Mantecon, T
Article. 10.1016/j.nrleng.2018.03.023. 2021
ANKK1 is found in myogenic precursors and muscle fibers subtypes with glycolytic metabolism
Rubio-Solsona, E; (...); Hoenicka, J
Article. 10.1371/journal.pone.0197254. 2018
Antibodies against nodo-paranodal proteins are not present in genetic neuropathies.
Martín-Aguilar L; (...); Querol L
Article. 10.1212/WNL.0000000000009189. 2020
Antimicrobial stewardship programme implementation in a medical ward
Ruiz, J; (...); Poveda, JL
Article. 2018
Antimicrobial stewardship programme in critical care medicine: A prospective interventional study
Ruiz, J; (...); Catellanos, A
Article. 10.1016/j.medin.2017.07.002. 2018
AntimiR treatment corrects myotonic dystrophy primary cell defects across several CTG repeat expansions with a dual mechanism of action
Cerro-Herreros, Estefania; (...); Artero, Ruben
Article. 10.1126/sciadv.adn6525. 2024
Assessing the value of moderate-to-severe atopic dermatitis treatment using multi-criteria decision analysis (MCDA).
Pereyra-Rodríguez JJ; (...); Ortiz de Frutos FJ
Letter. 10.1111/jdv.19432. 2023
Asymptomatic HyperCKemia in the Pediatric Population A Prospective Study Utilizing Next-Generation Sequencing and Ancillary Tests
Marti, Pilar; (...); Vilchez, Juan Jesus
Article. 10.1212/WNL.0000000000210116. 2025
Atypical desminopathy with new mutation and autosomal recessive transmission
Marti, P; (...); Padilla, JJV
Meeting Abstract. 2018
Baricitinib against severe COVID-19: effectiveness and safety in hospitalised pretreated patients.
Iglesias Gómez R; (...); Poveda Andrés JL
Article. 10.1136/ejhpharm-2021-002741. 2021
Bayesian pharmacokinetic individualization of prophylaxis with recombinant factor VIII in severe or moderate hemophilia A
Megias-Vericat, JE; (...); Bonanad, S
Meeting Abstract. 2018
Bayesian pharmacokinetic-guided prophylaxis with recombinant factor VIII in severe or moderate haemophilia A
Megias-Vericat, JE; (...); Poveda, JL
Article. 10.1016/j.thromres.2018.12.027. 2019
Bi-allelic mutations in EGR2 cause autosomal recessive demyelinating neuropathy by disrupting the EGR2-NAB complex.
Article. 10.1111/ene.14512. 2020
Bioavailability of once-daily tacrolimus formulations used in clinical practice in the management of De Novo kidney transplant recipients: the better study.
Fernandez Rivera C; (...); Hernandez D
Article. 10.1111/ctr.14550. 2022
Cardiac phenotype in glycogen storage disease type XV: a rare cardiomyopathy to bear in mind.
Mancheño N; (...); Zorio, Esther
Letter. 10.1016/j.recesp.2020.05.033. 2021
Clinical and Genetic Analysis of Patients With TK2 Deficiency.
Ceballos F; (...); Domínguez-González C
Article. 10.1212/NXG.0000000000200138. 2024
Clinical and genetic characteristics of 21 Spanish patients with biallelic pathogenic SPG7 mutations.
Baviera-Muñoz R; (...); Espinós C
Article. 10.1016/j.jns.2021.118062. 2021
Clinical and genetic profile, and assessment of disability in childhood hereditary motor neuropathy
Argente-Escrig, H; (...); Menezes, M
Meeting Abstract. 2020
Clinical and genetic spectrum of a large cohort of delta-sarcoglycan muscular dystrophy
Alonso-Perez, J; (...); Diaz-Manera, J
Meeting Abstract. 10.1016/j.nmd.2021.07.202. 2021
Clinical and genetic spectrum of a large cohort of patients with delta-sarcoglycan muscular dystrophy.
Alonso-Perez, Jorge; (...); Diaz-Manera, Jordi
Article. 10.1093/brain/awab301. 2021
Clinical and Magnetic Resonance Imaging features of a series of 11 Spanish patients who carry mutations in the BICD2 gene
Carrera, MF; (...); Sevilla, T
Meeting Abstract. 2018
Clinical and therapeutic features of myasthenia gravis in adults based on age at onset.
Cortés-Vicente E; (...); Illa I
Article. 10.1212/WNL.0000000000008903. 2020
Clinical benefits of a Bayesian model for plasma-derived factor VIII/VWF after one year of pharmacokinetic-guided prophylaxis in severe/moderate hemophilia A patients
Megias-Vericat, JE; (...); Poveda, JL
Article. 10.1016/j.thromres.2021.07.009. 2021
Clinical characteristics and outcomes of thymoma associated myasthenia gravis.
Álvarez-Velasco R; (...); Cortés-Vicente E
Article. 10.1111/ene.14820. 2021
Clinical features, mutation spectrum and factors related to reaching molecular diagnosis in a cohort of patients with distal myopathies.
Muelas, Nuria; (...); Vilchez, Juan J
Article. 10.1007/s00415-024-12821-3. 2025
Clinical pharmacy services: from cost-effectiveness analysis to a productivity indicators model
Nogueras, A; (...); Calleja, MA
Letter. 10.1136/ejhpharm-2019-001905. 2019
Clinical, genetic and disability profile of pediatric distal hereditary motor neuropathy.
Argente-Escrig H; (...); Menezes MP
Article. 10.1212/WNL.0000000000011054. 2021
Clinical, Histological, and Genetic Features of 25 Patients with Autosomal Dominant Progressive External Ophthalmoplegia (ad-PEO)/PEO-Plus Due to TWNK Mutations.
Bermejo-Guerrero, Laura; (...); Martin, Miguel A.
Article. 10.3390/jcm11010022. 2021
Clinical, pathological and genetic spectrum in 89 cases of mitochondrial progressive external ophthalmoplegia
Rodriguez-Lopez, C; (...); Dominguez-Gonzalez, C
Article. 10.1136/jmedgenet-2019-106649. 2020
Collaborative model of home delivery program in haemophilia through an association of patients
Megias-Vericat, JE; (...); Bonanad, S
Meeting Abstract. 2018
Comparison of pharmacokinetic estimates using generic and specific population pharmacokinetic models of plasma-derived factor VIII/VWF using WAPPS-Hemo
Megias-Vericat, JE; (...); Poveda, JL
Meeting Abstract. 2020
Consensus on the diagnosis, treatment and follow-up of patients with Duchenne muscular dystrophy.
Nascimento Osorio A; (...); Vilchez Padilla JJ
Article. 10.1016/j.nrl.2018.01.001. 2019
Consensus recommendations for the improvement of inter- and intra-centre care coordination in the management of hemophilia.
Montoro-Ronsano JB; (...); Soto-Ortega I
Article. 10.1016/j.farma.2023.01.003. 2023
Consensus recommendations for the improvement of inter- and intra-centre care coordination in the management of hemophilia.
Montoro-Ronsano, Jose Bruno; (...); Soto-Ortega, Inmaculada
Article. 10.1016/j.farma.2023.04.002. 2023
Correction to: Analysis of muscle magnetic resonance imaging of a large cohort of patient with VCP-mediated disease reveals characteristic features useful for diagnosis.
Esteller D; (...); Díaz-Manera J
Correction. 10.1007/s00415-023-12178-z. 2024
COST MINIMIZATION ANALYSIS OF RITUXIMAB BIOSIMILAR VERSUS REFERENCE RITUXIMAB IN THE TREATMENT OF FOLLICULAR LYMPHOMA
Gironella, M.; (...); Oyaguez, I
Meeting Abstract. 2019
COST-EFFECTIVENESS ANALYSIS OF DIFELIKEFALIN FOR THE TREATMENT OF CHRONIC KIDNEY DISEASE ASSOCIATED-PRURITUS IN SPAIN
Sanchez-Alvarez, Emilio E.; (...); Hernaez Colque, M.
Meeting Abstract. 2024
Cost-effectiveness of Cladribine Tablets and fingolimod in the treatment of relapsing multiple sclerosis with high disease activity in Spain.
Poveda JL; (...); Torres C
Article. 10.1080/14737167.2019.1635014. 2020
COST-EFFECTIVENESS OF CLADRIBINE TABLETS AND FINGOLIMOD IN THE TREATMENT OF REMITTING MULTIPLE SCLEROSIS WITH HIGH DISEASE ACTIVITY IN SPAIN
Trillo, JL; (...); Torres, C
Meeting Abstract. 10.1016/j.jval.2018.09.2029. 2018
COSTS OF THE MANAGEMENT OF HEMOPHILIA A WITH INHIBITORS IN SPAIN
Bonanad, S; (...); Gonzalez-Dominguez, A
Meeting Abstract. 2020
Costs of the management of hemophilia A with inhibitors in Spain
Bonanad, S; (...); Gonzalez-Dominguez, A
Article. 10.33393/grhta.2021.2234. 2021
CRISPR-Cas9 editing of a TNPO3 mutation in a muscle cell model of limb-girdle muscular dystrophy type D2.
Poyatos-Garcia, Javier; (...); Vilchez, Juan Jesus
Article. 10.1016/j.omtn.2023.01.004. 2023
Cross-sectional comparative study of pharmacokinetics and efficacy between sucrose-formulated recombinant factor VIII (Kogenate (R)) and BAY 81-8973 (KovaltryA (R)) in patients with severe or moderate haemophilia A in prophylaxis
Megias-Vericat, JE; (...); Poveda, JL
Letter. 10.1111/hae.13733. 2019
Cross-Sectional Comparative Study of PK-Guided Switch between Standard Half-Life and Extended Half-Life Factor VIII Products
Eduardo Megias, Juan; (...); Iorio, Alfonso
Meeting Abstract. 10.1182/blood-2019-127946. 2019
Cybersecurity, a priority for pharmacy services in the age of artificial intelligence.
Hernández Marín CM, Monte-Boquet E, Poveda Andrés JL
Editorial Material. 10.1016/j.farma.2024.08.001. 2024
Characterising the phenotype and mode of inheritance of patients with inherited peripheral neuropathies carrying MME mutations
Lupo, V; (...); SEVILLA, T
Article. 10.1136/jmedgenet-2018-105650. 2018
Characterization of molecular mechanisms underlying the axonal Charcot-Marie-Tooth neuropathy caused by MORC2 mutations
Sancho, P; (...); Espinos, C
Article. 10.1093/hmg/ddz006. 2019
Characterizing SOD1 mutations in Spain. The impact of genotype, age, and sex in the natural history of the disease.
Vazquez-Costa, Juan F.; (...); Garcia-Redondo, Alberto
Article. 10.1111/ene.15661. 2022
Charcot-Marie-Tooth disease due to MORC2 mutations in Spain
Sivera R; (...); Sevilla T
Article. 10.1111/ene.15001. 2021
Daunorubicin and cytarabine for certain types of poor-prognosis acute myeloid leukemia: a systematic literature review
Megias-Vericat, JE; (...); Montesinos, P
Review. 10.1080/17512433.2019.1573668. 2019
Defining a set of standardised outcome measures for newly diagnosed patients with multiple myeloma using the Delphi consensus method: the IMPORTA project
Blade, J; (...); Lizan, L
Article. 10.1136/bmjopen-2017-018850. 2018
Deletion of exons 45 to 55 in the DMD gene: from the therapeutic perspective to the in vitro model
Poyatos-Garcia, Javier; (...); Vilchez, Juan J
Article. 10.1186/s13395-024-00353-3. 2024
DEMOGRAPHICS AND TREATMENT PATTERNS FOR CHARCOT-MARIE-TOOTH DISEASE IN THE EU AND US: INTERIM RESULTS FROM AN INTERNATIONAL DIGITAL REAL-WORLD STUDY
Ziemssen, T.; (...); Larkin, M.
Meeting Abstract. 2019
DEPRESSION IN PATIENTS WITH CHARCOT-MARIE-TOOTH DISEASE TYPE 1A (CMT1A): FINDINGS FROM A REAL-WORLD DIGITAL STUDY
Thomas, Florian; (...); Boutalbi, Youcef
Meeting Abstract. 2022
Description of clinical and genetic features of 122 patients included in the Spanish Pompe registry
Martinez-Marin, Rafael Jenaro; (...); Diaz-Manera, Jordi
Article. 10.1016/j.nmd.2023.10.001. 2024
Design and rationale for a real-world prospective, multicenter registry of myocardial revascularization failure and secondary revascularization: The REVASEC study.
Salinas P; (...); Escaned J
Article. 10.1016/j.carrev.2021.11.015. 2021
Design of a Phase 2, Randomized, Double-Blind, Placebo-Controlled, 24-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Subjects with Facioscapulohumeral Muscular Dystrophy (FSHD): ReDUX4
Tawil, A; (...); Cadavid, D
Meeting Abstract. 2020
Determining the value contribution of emicizumab (Hemlibra (R)) for the prophylaxis of haemophilia A with inhibitors in Spain by multi-criteria decision analysis
Alvarez-Roman, MT; (...); Badia, X
Article. 10.1177/2284240319880534. 2019
DETERMINING THE VALUE OF EMICIZUMAB FOR THE PROPHYLAXIS OF SEVERE HAEMOPHILIA A PATIENTS WITHOUT INHIBITORS IN SPAIN BY MULTI-CRITERIA DECISION ANALYSIS (MCDA)
Alvarez, E.; (...); Badia, F. J.
Meeting Abstract. 2019
Determining value in the treatment of activated PI3Kdelta syndrome in Spain: a multicriteria decision analysis from the perspective of key stakeholders.
Abad, Maria Reyes; (...); Gil, Alicia
Article. 10.33393/grhta.2024.3041. 2024
Determining what represents value in the treatment of prurigo nodularis and its key unmet needs in Spain through Multi-Criteria Decision Analysis
Silvestre, Juan Francisco; (...); Serra-Baldrich, Esther
Article. 10.1002/jvc2.275. 2024
DEVELOPMENT AND VALIDATION OF A MCDA FRAMEWORK FOR EVALUATION AND DECISION-MAKING OF ORPHAN DRUGS IN SPAIN
Poveda, J. L.; (...); Vitoria, I
Meeting Abstract. 2019
Development and validation of an MCDA framework for evaluation and decision-making of orphan drugs in Spain
Badia, X; (...); Poved, JL
Article. 10.1080/21678707.2019.1652163. 2019
Diagnoses in HyperCKemia: study from classical aproach to the use of new technologies (NGS).
Marti, P; (...); Padilla, JV
Meeting Abstract. 2018
Diagnostic delay and pathway analysis in paediatric Charcot-Marie-Tooth disease
Argente-Escrig, H; (...); Mantecon, MTS
Meeting Abstract. 2019
Diagnostic Efficacy of Genetic Studies in a Series of Hereditary Cerebellar Ataxias in Eastern Spain.
Baviera-Munoz, Raquel; (...); Aller, Elena
Article. 10.1212/NXG.0000000000200038. 2022
Diagnostic yield of an NGS panel of muscle genes in a Reference Unit in Neuromuscular Diseases
Marti, P; (...); Vilchez, J. J.
Meeting Abstract. 2020
Digenic inheritance involving a muscle-specific protein kinase and the giant titin protein causes a skeletal muscle myopathy.
Topf, Ana; (...); Straub, Volker
Article. 10.1038/s41588-023-01651-0. 2024
Disseminated toxoplasma infection after hematopoietic stem cell transplantation with myositis and encephalitis.
Asensi Cantó P; (...); Guerreiro M
Article. 10.1111/tid.14067. 2023
Distal hereditary motor neuropathies: mutation spectrum and genotype-phenotype correlation.
Frasquet, Marina; (...); Lupo, Vincenzo
Article. 10.1111/ene.14700. 2021
DRP2 mutations as a cause of Charcot Marie Tooth in Spain
Sivera, R.; (...); Sevilla, T.
Meeting Abstract. 2023
Drug-refractory myasthenia gravis: Clinical characteristics, treatments, and outcome.
Cortes-Vicente, Elena; (...); Gallardo, Eduard
Article. 10.1002/acn3.51492. 2022
Dystrophinopathic subjects with a specific mega-deletion of exons 45-55 in the DMD gene, as a template for CRISPR/Cas9 therapy in Duchenne muscular dystrophy
Poyatos-Garcia, J; (...); Vilchez, J
Meeting Abstract. 10.1016/j.nmd.2019.06.404. 2019
Dystrophinopathy Phenotypes and Modifying Factors in DMD Exon 45-55 Deletion
Poyatos-Garcia, Javier; (...); Jesus Vilchez, Juan
Article. 10.1002/ana.26461. 2022
Early Referral to an ALS Center Reduces Several Months the Diagnostic Delay: A Multicenter-Based Study.
Martínez-Molina M; (...); Vázquez-Costa JF
Article. 10.3389/fneur.2020.604922. 2020
Economic impact of optimising antiretroviral treatment in human immunodeficiency virus-infected adults with suppressed viral load in Spain, by implementing the grade A-1 evidence recommendations of the 2015 GESIDA/National AIDS Plan
Ribera, E; (...); Hernandez-Novoa, B
Article. 10.1016/j.eimc.2016.11.015. 2018
Eculizumab for a catastrophic relapse in NMOSD: case report.
Gorriz, David; (...); Casanova, Bonaventura
Article. 10.1007/s10072-023-06971-x. 2023
Efecto de la adición de fortificantes y de módulo de proteínas en la osmolalidad de la leche materna donada.
Torres Martínez E; (...); Vento M
Article. 10.1016/j.anpedi.2020.03.009. 2020
Effectiveness of Caplacizumab Nanobody in Acquired Thrombotic Thrombocytopenic Purpura Refractory to Conventional Treatment
Palanques-Pastor T; (...); Poveda Andrés JL
Article. 10.1159/000517813. 2021
Effects of Spinal Cord Stimulation in Patients with Small Fiber and Associated Comorbidities from Neuropathy After Multiple Etiologies
Canos-Verdecho, Angeles; (...); Morales-Suarez-Varela, Maria
Article. 10.3390/jcm14020652. 2025
eLearning of pharmacokinetic monitoring in hemophilia with application in the clinical practice
Bonanad, S; (...); Megias-Vericat, JE
Meeting Abstract. 2018
European Federation of the Neurological Societies guidelines on the diagnostic approach to paucisymptomatic or asymptomatic hyperCKemia.
Kyriakides T; (...); Hilton-Jones D
Letter. 10.1002/mus.26777. 2020
EVALUATING REAL LIFE CRITERIA THAT COULD DRIVE PRICING & REIMBURSEMENT (P&R) APPROVAL OF ORPHAN DRUGS (ODS) IN SPAIN
Badia, F. J.; (...); Shepherd, J.
Meeting Abstract. 2019
Expanding the Clinical Spectrum of DRP2-Associated Charcot-Marie-Tooth Disease.
Article. 10.1212/WNL.0000000000209174. 2024
Expanding the molecular characterization of the CMT2Z disease associated gene MORC2
Sancho, P; (...); Espinos, C
Meeting Abstract. 2019
Exploratory analysis for the implementation of antineoplastic logarithmic dose banding
Albert-Mari, A; (...); Poveda-Andres, JL
Article. 10.1007/s11096-018-0714-9. 2018
EXTENDED MULTICRITERIA DECISION ANALYSIS IN DIABETIC MACULAR EDEMA. MULTIDEX-EMD EXTENSION STUDY
F, de Andres-Nogales; (...); Martinez, M.
Meeting Abstract. 2019
Facial onset sensory and motor neuronopathy: a motor neuron disease with an oligogenic origin?
Vazquez-Costa, JF; (...); Sevilla, T
Article. 10.1080/21678421.2019.1582671. 2019
Family with a new mutation in the DES gene of autosomal recessive transmission
Marti, P; (...); Vilchez, J
Meeting Abstract. 10.1016/j.nmd.2018.06.299. 2018
Fecal Calprotectin Pretreatment and Induction Infliximab Levels for Prediction of Primary Nonresponse to Infliximab Therapy in Crohn's Disease
Beltran, B; (...); Nos, P
Article. 10.1159/000492626. 2019
Fetal exposure to tofacitinib during the first trimester: A healthy newborn case report
Fernandez-Sanchez, M; (...); Poveda-Andres, JL
Article. 10.1002/bdr2.1942. 2021
Genetic Variants Associated with Biological Treatment Response in Inflammatory Bowel Disease: A Systematic Review.
Plaza J; (...); Moret-Tatay I
Article. 10.3390/ijms25073717. 2024
Genotype and phenotype spectrum of SORD neuropathy
Cortese, A; (...); SORD Nat Hist Study Grp
Meeting Abstract. 2021
Genotype-phenotype correlations in recessive titinopathies.
Savarese M; (...); Udd B
Article. 10.1038/s41436-020-0914-2. 2020
Genotype-phenotype correlations in valosin-containing protein disease: a retrospective muticentre study.
Schiava, Marianela; (...); Diaz-Manera, Jordi
Article. 10.1136/jnnp-2022-328921. 2022
Givinostat in DMD: results of the Epidys Study
Mercuri, E.; (...); McDonald, C.
Meeting Abstract. 10.1016/j.nmd.2022.07.223. 2022
Guide to Good Humanization Practices in Patients with Rare Diseases. who vadis
Editorial Material. 2022
Hazardous drugs levels in compounding area surfaces of Hospital Pharmacies Services: multicentric study
Valero-Garcia, S; (...); Spanish Hosp Pharmacy Units
Article. 10.7399/fh.10935. 2018
HEALTHCARE RESOURCE USE IN CHARCOT-MARIE-TOOTH DISEASE IN THE EU AND US: INTERIM RESULTS FROM AN INTERNATIONAL DIGITAL REAL-WORLD STUDY
Ziemssen, T.; (...); Larkin, M.
Meeting Abstract. 2019
Healthcare Resource Utilization among Patients between 60-75 Years with Secondary Acute Myeloid Leukemia Receiving Intensive Chemotherapy Induction: A Spanish Retrospective Observational Study
Solana-Altabella, Antonio; (...); Martinez-Cuadron, David
Article. 10.3390/cancers14081921. 2022
Hereditary transthyretin amyloidosis caused by the Val142Ile variant in Spain.
de Frutos, Fernando; (...); Gonzalez-Costello, Jose
Article. 10.1016/j.rec.2024.12.012. 2025
Hereditary transthyretin amyloidosis caused by Val142Ile variant in Spain: phenotypic characteristics, geographic distribution and population frequency
Seminario, F. De Frutos; (...); Gonzalez-Costello, J.
Meeting Abstract. 10.1093/eurheartj/ehae666.2073. 2024
High incidence of pulmonary thromboembolism in hospitalized SARS-CoV-2 infected patients despite thrombo-prophylaxis.
El-Qutob D; (...); Carrera-Hueso FJ
Article. 10.1016/j.hrtlng.2022.02.003. 2022
HNRNPDL-related limb girdle muscular dystrophy in a Spanish family with scapulo-peroneal phenotype, the first family in Europe
Vicente, LM; (...); VILCHEZ, JJ
Letter. 10.1016/j.jns.2020.116875. 2020
Hospital pharmacy initiatives for improving the management of patients with congenital coagulopathies
Bruno Montoro-Ronsano, Jose; (...); Alberto Vella, Vincenzo
Article. 10.7399/fh.11729. 2022
Hospitalization budget impact during the COVID-19 pandemic in Spain.
Carrera-Hueso, F. J.; (...); Crespo-Palomo, C.
Article. 10.1186/s13561-021-00340-0. 2021
How can artificial intelligence optimize value-based contracting?
Poveda, Jose Luis; (...); Medrano, Ignacio H.
Letter. 10.1186/s40545-022-00475-3. 2022
Human skeletal myopathy myosin mutations disrupt myosin head sequestration
Carrington, Glenn; (...); Ochala, Julien
Article. 2023
Human skeletal myopathy myosin mutations disrupt myosin head sequestration.
Carrington, Glenn; (...); Ochala, Julien
Article. 10.1172/jci.insight.172322. 2023
Identification of the missing causative mutations in monoallelic cases of late-onset Pompe disease (LOPD)
Gandia, M; (...); del Castillo, FJ
Meeting Abstract. 10.1016/j.ymgme.2019.11.138. 2020
Immune-mediated necrotising inflammatory myopathy. A rare side effect of statins.
Alarcon Chulilla, Mara; (...); Real Collado, Jose Tomas
Article. 10.1016/j.endien.2022.10.001. 2022
Impact of combinations of single-nucleotide polymorphisms of anthracycline transporter genes upon the efficacy and toxicity of induction chemotherapy in acute myeloid leukemia.
Megías-Vericat JE; (...); Montesinos P
Article. 10.1080/10428194.2020.1839650. 2020
Impact of NGS on HyperCKemia diagnosis
Marti, P; (...); Vilchez, J
Meeting Abstract. 10.1016/j.nmd.2019.06.506. 2019
Impact of SARS-CoV-2 infection and COVID-19 pandemic on the morbidity and mortality of amyotrophic lateral sclerosis patients in Valencia, Spain
Garcia-Casanova, Pilar H.; (...); Vazquez-Costa, Juan F.
Article. 10.1111/ene.16465. 2024
Impact of the therapeutic positioning report in the P&R process in Spain: analysis of orphan drugs approved by the European Commission and reimbursed in Spain from 2003 to 2019
Badia X; (...); Hernández C
Article. 10.1186/s13023-020-01507-4. 2020
Impact of voriconazole plasma concentrations on treatment response in critically ill patients
Ruiz, J; (...); Ramirez, P
Article. 10.1111/jcpt.12817. 2019
Implementation and results of a risk-sharing scheme for enzyme replacement therapy in lysosomal storage diseases
Edo-Solsona, MD, Vitoria-Minana, I, Poveda-Andres, JL
Article. 10.7399/fh.11262. 2020
Implication of the breakpoints position in patients with the macrodeletion of exons 45 to 55 in the DMD gene
Poyatos-Garcia, J; (...); Vilchez, JJ
Meeting Abstract. 2019
Influence of concomitant immunomodulators during maintenance therapy with Adalimumab in inflammatory bowel disease: looking for the ideal patient to use monotherapy
Bastida, G; (...); Nos, P
Meeting Abstract. 10.1093/ecco-jcc/jjy222.581. 2019
Influence of pharmacokinetic/pharmacodynamic ratio on vancomycin treatment response in paediatric patients with Staphylococcus aureus bacteremia.
Ruiz J; (...); Poveda JL
Article. 10.23736/S0026-4946.18.04978-2. 2018
Influence of polymorphisms in anthracyclines metabolism genes in the standard induction chemotherapy of acute myeloid leukemia.
Megías-Vericat JE; (...); Montesinos P
Article. 10.1097/FPC.0000000000000431. 2021
Influence of the intronic breakpoint of the DYS 45-55 exon deletion on the clinical phenotype
Poyatos, J; (...); Vilchez, J
Meeting Abstract. 10.1016/j.nmd.2018.06.270. 2018
Insights into phenotypic variability caused by GARS1 pathogenic variants.
Jiménez-Jiménez J; (...); Sivera R
Article. 10.1111/ene.16416. 2024
Intraperitoneal vancomycin in neonates during peritoneal dialysis: A case report
Company-Albir, MJ; (...); Poveda, JL
Article. 10.1111/jcpt.12842. 2019
Intravitreal melphalan therapy for vitreous seeds in retinoblastoma: Implementation and outcomes of a new chemotherapy protocol.
Solana-Altabella A; (...); Poveda JL
Article. 10.1177/1078155220904410. 2020
Involvement of interleukin 6 in SARS-CoV-2 infection: siltuximab as a therapeutic option against COVID-19
Palanques-Pastor, T, Lopez-Briz, E, Andres, JLP
Article. 10.1136/ejhpharm-2020-002322. 2020
IqYmune® is an effective maintenance treatment for multifocal motor neuropathy: a randomised, double-blind, multicenter cross-over non-inferiority study versus Kiovig® - The LIME Study.
Léger JM; (...); Merkies I
Article. 10.1111/jns.12291. 2019
Is erenumab an efficient alternative for the prevention of episodic and chronic migraine in Spain? Results of a cost-effectiveness analysis.
Pozo-Rosich P; (...); Irimia P
Article. 10.1186/s10194-024-01747-w. 2024
Isavuconazole Pharmacokinetics in Critically Ill Patients: Relationship with Clinical Effectiveness and Patient Safety.
Martin-Cerezuela, Maria; (...); Ramirez Galleymore, Paula
Article. 10.3390/antibiotics13080706. 2024
ITPR3-associated neuropathy: Report of a further family with adult onset intermediate Charcot-Marie-Tooth disease.
Cabello-Murgui, Javier; (...); Sivera, Rafael
Article. 10.1111/ene.16485. 2024
KY mutations are a cause of distal neuromyopathies
Muelas, N.; (...); Vilchez, J.
Meeting Abstract. 2023
Lenalidomide induced reversible parkinsonism, dystonia, and dementia in subclinical Creutzfeldt-Jakob disease
Argente-Escrig, H; (...); Bataller, L
Letter. 10.1016/j.jns.2018.08.027. 2018
Management of flexible cavovarus foot in patients with Charcot-Marie-Tooth disease: midterm results.
Megías-Vericat JE; (...); Poveda-Andrés JL
Article. 2021
Management of Hereditary Transthyretin Amyloidosis (ATTRv) Patients and Asymptomatic Carriers in Spain: The EMPATIa Study.
Losada Lopez, Ines; (...); Setaro, Francesca
Article. 10.3390/jcm13247587. 2024
Manipulación de fármacos para su administración por gastrostomía endoscópica percutánea en pacientes con esclerosis lateral amiotrófica y nutrición enteral.
Vázquez Polo A; (...); Vázquez Costa JF
Article. 10.20960/nh.03946. 2022
Matching-Adjusted Indirect Comparison of Efficacy and Consumption of rVIII-SingleChain Versus Two Recombinant FVIII Products Used for Prophylactic Treatment of Adults/Adolescents with Severe Haemophilia A
Bonanad, S; (...); Santos, S
Article. 10.1007/s12325-021-01853-0. 2021
Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy
Campbell, C; (...); Clinical Evaluator Training Grp
Article. 10.2217/cer-2020-0095. 2020
Metabolic Phenotyping in Prostate Cancer Using Multi-Omics Approaches.
Gomez-Cebrian, Nuria; (...); Puchades-Carrasco, Leonor
Article. 10.3390/cancers14030596. 2022
Metrics of progression and prognosis in untreated adults with thymidine kinase 2 deficiency: An observational study.
Dominguez-Gonzalez, Cristina; (...); Martin, Miguel Angel
Article. 10.1016/j.nmd.2022.07.399. 2022
Metyrapone as treatment in the neonatal McCune-Albright syndrome
de Mingo, Carmen; (...); Garcia-Robles, Ana
Article. 10.1515/jpem-2020-0036. 2020
Mitochondrial developmental encephalopathy with bilateral optic neuropathy related to homozygous variants in IMMT gene.
Marco-Hernández AV; (...); Martínez-Castellano F
Article. 10.1111/cge.14093. 2022
Molecular diagnosis of inherited peripheral neuropathies: gene panel vs. exome sequencing
Lupo, V; (...); Espinos, C
Meeting Abstract. 2018
Monitoring coagulation factors during surgery. A systematic review
Eduardo Megias-Vericat, Juan; (...); Luis Poveda-Andres, Jose
Review. 10.7399/fh.11766. 2021
Monitoring contamination of hazardous drug compounding surfaces at hospital pharmacy departments. A consensus Statement. Practice guidelines of the Spanish Society of Hospital Pharmacists (SEFH).
Valero-García S; (...); Delgado-Sánchez O
Article. 10.7399/fh.11655. 2021
Monitoring the quality of the hospital pharmacoterapeutic process by sentinel patient program.
Fernandez-Megia, MJ; (...); Poveda Andrés JL
Article. 10.7399/fh.10793. 2018
MRI prognostic outcome in asymptomatic/paucisymptomatic HyperCKemia
Marti, P; (...); Padilla, JV
Meeting Abstract. 2018
Msi2 enhances muscle dysfunction in a myotonic dystrophy type 1 mouse model.
Sabater-Arcis, Maria; (...); Artero, Ruben
Article. 10.1016/j.bj.2023.100667. 2023
Multi-Omic Approaches to Breast Cancer Metabolic Phenotyping: Applications in Diagnosis, Prognosis, and the Development of Novel Treatments
Gomez-Cebrian, N; (...); Pineda-Lucena, A
Review. 10.3390/cancers13184544. 2021
Muscle MRI characteristic pattern for late-onset TK2 deficiency diagnosis
Dominguez-Gonzalez, C; (...); Paradas, C
Meeting Abstract. 10.1016/j.nmd.2021.07.358. 2021
Muscle MRI characteristic pattern for late-onset TK2 deficiency diagnosis.
Dominguez-Gonzalez, Cristina; (...); Paradas, Carmen
Article. 10.1007/s00415-021-10957-0. 2022
Muscle MRI in a large cohort of patients with oculopharyngeal muscular dystrophy
Alonso-Jimenez, A; (...); Diaz-Manera, J
Article. 10.1136/jnnp-2018-319578. 2019
Muscle MRI-phenotyping of patients with likely pathogenic anoctamin 5 variants
Poulsen, N.; (...); Vissing, J.
Meeting Abstract. 10.1016/j.nmd.2024.07.187. 2024
Mutations in MME cause autosomal recessive late-onset CMT type-2 disease
Carrera, MF; (...); Sevilla, T
Meeting Abstract. 2018
Myosin ATPase inhibition fails to rescue the metabolically dysregulated proteome of nebulin-deficient muscle.
Laitila, Jenni; (...); Ochala, Julien
Article. 10.1113/JP286870. 2024
Myosin dysregulation in nemaline myopathy
Laitila, J.; (...); Ochala, J.
Article. 10.1016/j.nmd.2022.07.017. 2022
National Survey of the SEFH-2019: portfolio of services, healthcare activity, teaching and research in Hospital Pharmacy Services in Spain (vol 45, pg 32, 2021)
Perez-Encinas, Montserrat; (...); Angel Calleja-Hernandez, Miguel
Correction. 10.7399/fh.11740. 2021
National Survey on the Use of Mobile Applications in Patients with Hemophilia and Other Coagulopathies
Eduardo Megias, Juan; (...); Poveda Andres, Jose Luis
Meeting Abstract. 10.1182/blood-2022-169938. 2022
NATURAL HISTORY STUDY OF SORD NEUROPATHY
Cortese, Andrea; (...); Zuchner, Stephan
Meeting Abstract. 2022
NEB mutations disrupt the super-relaxed state of myosin and remodel the muscle metabolic proteome in nemaline myopathy.
Ranu, Natasha; (...); Ochala, Julien
Article. 10.1186/s40478-022-01491-9. 2023
New challenges of advanced therapies.
Megías-Vericat JE; (...); Poveda Andrés JL
Article. 10.1016/j.farma.2023.11.008. 2024
New developments and data highlights in the international myotubular and centronuclear myopathy patient registry
Laitila, J.; (...); Ochala, J.
Meeting Abstract. 10.1016/j.nmd.2022.07.018. 2022
New insights into the pathophysiology of fasciculations in amyotrophic lateral sclerosis: An ultrasound study
Vazquez-Costa, JF; (...); Sevilla, T
Article. 10.1016/j.clinph.2018.09.014. 2018
New mutations found by Next-Generation Sequencing screening of Spanish patients with Nemaline Myopathy
Moreau-Le Lan, S; (...); Pedrola, L
Article. 10.1371/journal.pone.0207296. 2018
Noninfectious Neurologic Complications after Allogeneic Hematopoietic Stem Cell Transplantation
Balaguer-Rosello, A; (...); Sanz, Jaime
Article. 10.1016/j.bbmt.2019.05.024. 2019
Nusinersen in adult patients with 5q spinal muscular atrophy: Amulticenter observational cohorts' study.
Vazquez-Costa, Juan F.; (...); Hervas, David
Article. 10.1111/ene.15501. 2022
Observational study to evaluate discontinuation of monotherapy with cobicistat-boosted darunavir in patients with human immunodeficiency virus
Solana-Altabella, Antonio; (...); Poveda-Andres, Jose Luis
Article. 10.1097/MD.0000000000032208. 2022
ODs with a positive TPR conclusion, not subject to a conditional approval, and approved without requering a pass would be more likely to be reimbursed in Spain.
Poveda JL; (...); Badia X
Article. 10.1186/s13023-022-02610-4. 2023
Omics-Based Approaches for the Characterization of Pompe Disease Metabolic Phenotypes
Gomez-Cebrian, Nuria; (...); Puchades-Carrasco, Leonor
Review. 10.3390/biology12091159. 2023
Onasemnogene Abeparvovec Administration via Peripherally Inserted Central Catheter: A Case Report.
Pitarch Castellano, Inmaculada; (...); Poveda Andres, Jose L
Case Reports. 10.3390/children11050590. 2024
OPTIMIZING MANAGEMENT AND OUTCOMES OF ACTIVATED PHOSPHOINOSITIDE 3-KINASE DELTA SYNDROME (APDS) IN SPAIN: BEWAYAPDS PROJECT
Flores-Moreno, S.; (...); Gil, A.
Meeting Abstract. 2024
Outcome of patients with sustained hyperCKemia after statin treatment
Muelas, N; (...); Vilchez, JJ
Meeting Abstract. 2019
Oxidative Stress, a Crossroad between Rare Diseases and Neurodegeneration
Espinos, C; (...); Pallardo, FV
Review. 10.3390/antiox9040313. 2020
Paracetamol vs. Ibuprofen in Preterm Infants With Hemodynamically Significant Patent Ductus Arteriosus: A Non-inferiority Randomized Clinical Trial Protocol
Garcia-Robles, Ana; (...); Carrascosa, MA
Article. 10.3389/fped.2020.00372. 2020
Paracetamol vs. Ibuprofen in Preterm Infants With Hemodynamically Significant Patent Ductus Arteriosus: A Non-inferiority Randomized Clinical Trial Protocol (vol 8, 372, 2020)
Garcia-Robles, Ana; (...); Aguar Carrascosa, Marta
Correction. 10.3389/fped.2021.834454. 2022
Patient and health practitioner perceptions about the role of hospital pharmacists along the care continuum: the Fharmaconectados Project
Morillo-Verdugo, R; (...); Diaz-Olmo, J
Article. 10.7399/fh.11719. 2021
Patient and health practitioner perceptions about the role of hospital pharmacists along the care continuum: the Fharmaconectados Project.
Morillo-Verdugo R; (...); Díaz-Olmo J
Article. 2021
Patient-reported impact of Charcot-Marie-Tooth disease: protocol for a real-world digital lifestyle study.
Thomas, Florian P.; (...); Boutalbi, Youcef
Review. 10.2217/nmt-2020-0044. 2021
PATIENT-REPORTED SYMPTOM BURDEN OF CHARCOT-MARIE-TOOTH DISEASE TYPE 1A (CMT1A): FINDINGS FROM A REAL-WORLD DIGITAL STUDY
Thomas, Florian; (...); Boutalbi, Youcef
Meeting Abstract. 2022
Patient-Reported Symptom Burden of Charcot-Marie-Tooth Disease Type 1A: Findings From an Observational Digital Lifestyle Study.
Thomas, Florian P; (...); Boutalbi, Youcef
Article. 10.1097/CND.0000000000000426. 2022
Pediatric inherited peripheral neuropathy: a prospective study at a Spanish referral center
Argente-Escrig H; (...); Sevilla T
Article. 10.1002/acn3.51432. 2021
Persistent asymptomatic or mild symptomatic hyperCKemia due to mutations in ANO5: the mildest end of the anoctaminopathies spectrum
Panades-de Oliveira, L; (...); Dominguez-Gonzalez, C
Article. 10.1007/s00415-020-09872-7. 2020
Pharmaceutical care to hospital outpatients during the COVID-19 pandemic. Telepharmacy
Margusino-Framiñán L; (...); Morillo-Verdugo R
Article. 10.7399/fh.11498. 2020
Pharmacogenetics in Response to Biological Agents in Inflammatory Bowel Disease: A Systematic Review.
Ballesta-Lopez, Octavio; (...); Poveda-Andres, Jose Luis
Article. 10.3390/ijms26041760. 2025
Pharmacometabolomics by NMR in Oncology: A Systematic Review.
Gómez-Cebrián N; (...); Pineda-Lucena A
Article. 10.3390/ph14101015. 2021
Pharmacotherapeutic management of advanced therapy medicinal products
Luis Poveda-Andres, Jose; (...); Valero-Garcia, Silvia
Article. 10.7399/fh.13036. 2022
Phenotype and clinical outcomes of Glu89Lys hereditary transthyretin amyloidosis: a new endemic variant in Spain.
de Frutos, Fernando; (...); Garcia-Pavia, Pablo
Article. 10.1080/13506129.2022.2142110. 2022
Phenotypical features of two patients diagnosed with PHARC syndrome and carriers of a new homozygous mutation in the ABHD12 gene
Frasquet, M; (...); Sevilla, T
Article. 10.1016/j.jns.2018.02.021. 2018
Pilot evaluation of home delivery programme in haemophilia
Megias-Vericat, JE; (...); Poveda-Andres, JL
Article. 10.1111/jcpt.12718. 2018
PK-GUIDED SWITCH BETWEEN STANDARD HALF-LIFE AND EXTENDED HALF-LIFE FACTOR VIII PRODUCTS
Vericat, JEM; (...); Iorio, A
Meeting Abstract. 2020
Plectin-related scapuloperoneal myopathy with treatment-responsive myasthenic syndrome.
Argente-Escrig H; (...); Schröder R
Letter. 10.1111/nan.12652. 2020
POGLUT1 biallelic mutations cause myopathy with reduced satellite cells, a-dystroglycan hypoglycosylation and a distinctive radiological pattern.
Servián-Morilla E; (...); Paradas C
Article. 10.1007/s00401-019-02117-6. 2020
Pre and post intervention study of antiblastic drugs contamination surface levels at a Pharmacy Department Compounding Area using a closed system drug transfer device and a decontamination process
Valero, S; (...); Poveda, JL
Article. 10.1016/j.yrtph.2018.03.001. 2018
Preclinical characterization of antagomiR-218 as a potential treatment for myotonic dystrophy.
Cerro-Herreros, E; (...); Artero, R
Article. 10.1016/j.omtn.2021.07.017. 2021
PRELIMINAR STUDY OF THERAPY ADHERENCE CONTROLLED BY PRESCRIPTION OF MEDICATION AND SELF-PERCEPTION ADHERENCE OF INFLAMMATORY CHRONIC DISEASE PATIENTS
Garcia, EG; (...); Ivorra, JAR
Meeting Abstract. 10.1136/annrheumdis-2019-eular.5937. 2019
Preparación y administración de perfusiones de insulina en neonatología: Encuesta nacional.
García-Robles A; (...); Aguar-Carrascosa M
Letter. 10.1016/j.anpedi.2020.06.008. 2021
Preparation and administration of insulin infusions in neonatology: A national survey.
Garcia-Robles, Ana; (...); Aguar Carrascosa, Marta
Article. 10.1016/j.anpede.2020.06.015. 2021
Presence of good humanization practices in the healthcare of patients with rare diseases in Pharmacy Services
Company Albir MJ, Poveda Andrés JL, Edo Solsona MD
Article. 10.1016/j.farma.2023.06.0131130. 2024
Presenilin-1 Mutations Are a Cause of Primary Lateral Sclerosis-Like Syndrome
Vazquez-Costa, JF; (...); SEVILLA, T
Article. 10.3389/fnmol.2021.721047. 2021
Proactive therapeutic drug monitoring and pharmacogenetic analysis in inflammatory bowel disease: A systematic review
Ballesta-Lopez, Octavio; (...); Luis Poveda-Andres, Jose
Review. 10.7399/fh.11780. 2021
PRODUCTIVITY LOSSES IN PEOPLE WITH CHARCOT-MARIE-TOOTH DISEASE IN THE EU AND US: INTERIM RESULTS FROM AN INTERNATIONAL DIGITAL REAL-WORLD EVIDENCE STUDY
Ziemssen, T.; (...); Larkin, M.
Meeting Abstract. 2019
Quantitative magnetic resonance imaging assessment of muscle composition in myotonic dystrophy mice
Bargiela, Ariadna; (...); Artero, Ruben
Article. 10.1038/s41598-023-27661-w. 2023
Randomized phase 2 study of ACE-083, a muscle-promoting agent, in facioscapulohumeral muscular dystrophy.
Statland, Jeffrey M.; (...); Attie, Kenneth M.
Article. 10.1002/mus.27558. 2022
Real life experience of tafamidis for the treatment of Spanish patients with Val30Met transthyretin amyloidosis with polyneuropathy.
Sanso, Maria Antonia Ribot; (...); Gonzalez-Moreno, Juan
Article. 10.1016/j.medcli.2024.01.008. 2024
Risdiplam in non-sitter patients aged 16years and older with 5q spinal muscular atrophy.
Nungo Garzon, Nancy Carolina; (...); Vazquez-Costa, Juan F
Article. 10.1002/mus.27804. 2023
Role of the nigrosome 1 absence as a biomarker in amyotrophic lateral sclerosis
Isabel Moreno-Gambin, Maria; (...); Vázquez-Costa JF
Article. 10.1007/s00415-021-10729-w. 2021
Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
Mercuri E; (...); McDonald CM
Article. 10.1016/S1474-4422(24)00036-X. 2024
Safety and efficacy of losmapimod in facioscapulohumeral muscular dystrophy (ReDUX4): a randomised, double-blind, placebo-controlled phase 2b trial.
Tawil R; (...); Jiang JG
Article. 10.1016/S1474-4422(24)00073-5. 2024
Safety and efficacy of nipocalimab in adults with generalised myasthenia gravis (Vivacity-MG3): a phase 3, randomised double-blind, placebo-controlled study
Antozzi, Carlo; (...); Sun, Hong
Article. 2025
Safety and Efficacy of Nipocalimab in Patients With Generalized Myasthenia Gravis: Results From the Randomized Phase 2 Vivacity-MG Study
Antozzi, Carlo; (...); Arroyo, Santiago
Article. 10.1212/WNL.0000000000207937. 2024
SEFH National Survey-2019: general characteristics, staffing, material resources and information systems in Spain's hospital pharmacy departments.
Pérez-Encinas M; (...); Calleja-Hernández MÁ
Article. 10.7399/fh.11565. 2020
SERUM AUTOANTIBODY LOWERING BY THE ANTI-FCRN MONOCLONAL ANTIBODY, NIPOCALIMAB, CORRELATES WITH CLINICAL IMPROVEMENT IN GENERALIZED MYASTHENIA GRAVIS PATIENTS
Guptill, Jeffrey; (...); Sun, Hong
Meeting Abstract. 2022
Short-term and long-term safety profile of Givinostat in Duchenne muscular dystrophy
Vucinic, D.; (...); Muelas, N.
Meeting Abstract. 10.1016/j.nmd.2024.07.289. 2024
SMPTall man lettering application in medication information systems as a quality and safety strategy in hospital organization
Iglesias Gomez, Ruben; (...); Poveda Andres, Jose Luis
Article. 10.1111/jcpt.13703. 2022
Spanish family with scapulo-peroneal myopathy due to HNRNPDL mutation: the first European family
Martinez Vicente, L.; (...); Vilchez, J. J.
Meeting Abstract. 2020
Spanish Pompe registry: analysis of the first 100 patients included
Reyes-Leiva, D; (...); S Spanish Pompe Study Grp
Meeting Abstract. 10.1016/j.nmd.2021.07.224. 2021
Spanish Pompe Registry: New data based on the 130 patients included
Marin, R. Martinez; (...); Manera, J. Diaz
Meeting Abstract. 2023
Spanish Pompe registry: new data based on the 130 patients included
Martinez Marin, R.; (...); Manera, J. Diaz
Meeting Abstract. 10.1016/j.nmd.2023.07.343. 2023
Spanish Pompe Registry: overview based on the 130 patients included
Marin, R. Martinez; (...); Manera, J. Diaz
Meeting Abstract. 10.1016/j.nmd.2024.07.732. 2024
Spanish Pompe Registry: Update of the 122 patients included
Martinez Marin, R.; (...); Diaz Manera, J.
Meeting Abstract. 10.1016/j.nmd.2022.07.145. 2022
Spinocerebellar Ataxia 36 is a Frequent Cause of Hereditary Ataxia in Eastern Spain
Baviera-Munoz, Raquel; (...); Bataller, Luis
Article. 10.1002/mdc3.13740. 2023
Statin-induced myopathies: beyond immuno-mediated necrotizing myopathies
Muelas, MMN; (...); Vilchez, JJ
Meeting Abstract. 2018
Status of the pivotal phase III study of PXT3003 for Charcot-Marie-Tooth Type 1A disease (CMT1A)
Goedkoop, R; (...); Cohen, D
Meeting Abstract. 2018
Systematic Review of Pharmacogenetics of ABC and SLC Transporter Genes in Acute Myeloid Leukemia
Megías-Vericat JE; (...); Montesinos P
Review. 10.3390/pharmaceutics14040878. 2022
Telemedicine in Neuromuscular Diseases During Covid-19 Pandemic: ERN-NMD European Survey.
El-Hassar L; (...); Sacconi S
Article. 10.3233/JND-221525. 2022
The association of salivary caffeine levels with serum concentrations in premature infants with apnea of prematurity
Garcia-Robles, Ana; (...); Saenz Gonzalez, Pilar
Article. 10.1007/s00431-022-04628-z. 2022
The contribution of fenfluramine to the treatment of Dravet syndrome in Spain through Multi-Criteria Decision Analysis.
Gil-Nagel A; (...); Gil A
Article. 10.1016/j.yebeh.2022.108711. 2022
The cross-sectional area of the median nerve: An independent prognostic biomarker in amyotrophic lateral sclerosis.
Martinez-Paya, J J; (...); Vazquez-Costa, J F
Article. 10.1016/j.nrleng.2024.07.003. 2024
The impact of rituximab infusion protocol on the long-term outcome in anti-MuSK myasthenia gravis
Cortes-Vicente, E; (...); Illa, I
Article. 10.1002/acn3.564. 2018
The mutation of Transportin 3 gene that causes limb girdle muscular dystrophy 1F induces protection against HIV-1 infection
Rodriguez-Mora, Sara; (...); Alcami, Jose
Article. 2019
The mutation of Transportin 3 gene that causes limb girdle muscular dystrophy 1F induces protection against HIV-1 infection.
Rodríguez-Mora S; (...); Alcamí J
Article. 10.1371/journal.ppat.1007958. 2019
The relationship between deficit in digit span and genotype in nonsense mutation Duchenne muscular dystrophy
Thangarajh, M; (...); Ataluren Phase 2b Study Grp
Article. 10.1212/WNL.0000000000006245. 2018
The value of the reflective discussion in decision-making using multi-criteria decision analysis (MCDA): an example of determining the value contribution of tabelecleucel for the treatment of the Epstein Barr virus-positive post-transplant lymphoproliferative disease (EBV+ PTLD).
Badia, Xavier; (...); Valles, Joan-Antoni
Article. 10.1186/s13023-024-03324-5. 2024
The view of experts on initiatives to be undertaken to promote equity in the access to orphan drugs and specialised care for rare diseases in Spain: A Delphi consensus
Torrent-Farnell, J; (...); Lizan, L
Article. 10.1016/j.healthpol.2018.03.002. 2018
The width of the third ventricle associates with cognition and behaviour in motor neuron disease
Vazquez-Costa, JF; (...); Sevilla, T
Article. 10.1111/ane.13022. 2019
Therapeutic potential of oleic acid supplementation in myotonic dystrophy muscle cell models.
Moreno, Nerea; (...); Artero, Ruben
Article. 10.1186/s40659-024-00496-z. 2024
Topiramate pharmacokinetics in neonates undergoing therapeutic hypothermia and proposal of an optimised dosing schedule.
Marques MR; (...); Mangas-Sanjuan V
Article. 10.1111/apa.14944. 2020
Towards a symbiotic relationship between big data, artificial intelligence, and hospital pharmacy
Del Rio-Bermudez, C; (...); Poveda, JL
Editorial Material. 10.1186/s40545-020-00276-6. 2020
Toxic-metabolic encephalopathy induced by metronidazole and disulfiram: classics never die.
Chovi-Trull, Maria; (...); Poveda-Andres, Jose Luis
Article. 10.1136/ejhpharm-2024-004184. 2024
Transcriptomic Evidence of the Immune Response Activation in Individuals With Limb Girdle Muscular Dystrophy Dominant 2 (LGMDD2) Contributes to Resistance to HIV-1 Infection
Diez-Fuertes F; (...); Rodríguez-Mora S
Article. 10.3389/fcell.2022.839813. 2022
Undetectable levels of adalimumab in clinical practice: Should we say goodbye to the drug?
Bejar, S; (...); Mateu, PN
Meeting Abstract. 2020
Use and handling safety of Mini-Spike 2 (R) chemo and puresite for safe chemotherapy compounding in a hospital pharmacy
Garcia, SV; (...); Andres, JLP
Article. 10.1016/j.yrtph.2018.10.002. 2018
USE OF CAPLACIZUMAB IN THE ACQUIRED THROMBOTIC THROMBOCYTOPENIC PURPURA
Dominguez Hernandez, L.; (...); Gomez Segui, I
Meeting Abstract. 2019
Use of NGS for diagnosis of asymptomatic hyperCKemia in childhood
Marti, P; (...); VILCHEZ, J
Meeting Abstract. 10.1016/j.nmd.2021.07.340. 2021
Validación de la versión española de la Charcot-Marie-Tooth Disease Pediatric Scale (CMTPedS).
Pitarch-Castellano I; (...); Burns J
Article. 10.33588/rn.7403.2021489. 2022
Validation of Neuromyotype: A smart keyboard for the evaluation of spinal muscular atrophy patients.
Cortes, P Lizandra; (...); Vázquez-Costa JF
Article. 10.1016/j.nrleng.2022.05.001. 2022
VALUE CRITERIA FOR OUTCOMES BASED MANAGED ENTRY AGREEMENT OF CAR-T IN RELAPSED OR REFRACTORY MULTIPLE MYELOMA (RRMM) THROUGH MCDA
Poveda, J. L.; (...); Lizan, L.
Meeting Abstract. 2023
Vamorolone versus placebo and prednisone in Duchenne muscular dystrophy: results from a 24-week double-blind randomized trial
Guglieri, M; (...); Andres Sant Joan Deu Hosp
Meeting Abstract. 2021
VIVACITY-MG: A PHASE 2, MULTICENTER, RANDOMIZED, DOUBLE- BLIND, PLACEBO-CONTROLLED STUDY TO EVALUATE THE SAFETY, TOLERABILITY, EFFICACY, PHARMACO-KINETICS, PHARMACODYNAMICS, AND IMMUNOGENICITY OF NIPOCALIMAB ADMINISTERED TO ADULTS WITH GENERALIZED MYASTHENIA GRAVIS
Antozzi, Carlo; (...); Arroyo, Santiago
Meeting Abstract. 2023
Vivacity-MG: A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Nipocalimab Administered to Adults with Generalized Myasthenia Gravis
Guptill, Jeffrey; (...); Arroyo, Santiago
Meeting Abstract. 2021
What is wrong with nuclei in Transportin 3 (TPNO3)-related muscular dystrophy?
Curtis-Wetton, E; (...); Ochala, J
Meeting Abstract. 10.1016/S0960-8966(18)30315-8. 2018
WORK IMPACTS IN PATIENTS WITH CHARCOT-MARIE-TOOTH DISEASE TYPE 1A (CMT1A): FINDINGS FROM A REAL-WORLD DIGITAL STUDY
Thomas, Florian; (...); Boutalbi, Youcef
Meeting Abstract. 2022