EFECTOS DE LEVOSIMENDÁN ORAL (ODM-109) EN LA FUNCIÓN RESPIRATORIA DE PACIENTES CON ELA: ESTUDIO DE EXTENSIÓN ABIERTO PARA PACIENTES QUE HAYAN COMPLETADO EL ESTUDIO 3119002.

Datos básicos

Código:
3119003
Protocolo:
3119003
EUDRACT:
2018-004180-31
NCT:
Centro:
HOSPITAL UNIVERSITARI I POLITÈCNIC LA FE
Dotación:
Año de incio:
2019
Año de finalización:
2023
ENSAYO CLÍNICO INTERNACIONAL

Documentos

  • No hay documentos

Participantes

Grupos

Financiadores - Promotores

ORION CORPORATION

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

  • Open Access.

[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

  • Open Access.

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 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 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 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


Advances in the early diagnosis of amyotrophic lateral sclerosis.

Garcia-Casanova, Pilar H, Vazquez-Costa, Juan F

Article. 10.1080/14737175.2025.2471556. 2025


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 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


Antibodies against nodo-paranodal proteins are not present in genetic neuropathies.

Martín-Aguilar L; (...); Querol L

Article. 10.1212/WNL.0000000000009189. 2020


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


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


Bi-allelic mutations in EGR2 cause autosomal recessive demyelinating neuropathy by disrupting the EGR2-NAB complex.

Lupo V; (...); Sevilla T

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


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 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 spectrum of BICD2 mutations.

Frasquet M; (...); Sevilla T

Article. 10.1111/ene.14173. 2020


Clinical trials in pediatric ALS: a TRICALS feasibility study.

Kliest T; (...); Van Den Berg LH

Article. 10.1080/21678421.2021.2024856. 2022


Clinical, genetic and disability profile of pediatric distal hereditary motor neuropathy.

Argente-Escrig H; (...); Menezes MP

Article. 10.1212/WNL.0000000000011054. 2021


CMT caused by MORC2 mutations in Spain

Sivera, R; (...); Sevilla, T

Meeting Abstract. 2020


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 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


COST MINIMIZATION ANALYSIS OF RITUXIMAB BIOSIMILAR VERSUS REFERENCE RITUXIMAB IN THE TREATMENT OF FOLLICULAR LYMPHOMA

Gironella, M.; (...); Oyaguez, I

Meeting Abstract. 2019

  • Open Access.

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


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


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


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


Delphi consensus on recommendations for the treatment of spinal muscular atrophy in Spain (RET-AME consensus)

Pitarch Castellano, I; (...); Vazquez-Costa, J. F.

Article. 10.1016/j.nrl.2021.07.008. 2022


Delphi consensus on recommendations for the treatment of spinal muscular atrophy in Spain (RET-AME consensus).

Pitarch Castellano I; (...); Vázquez-Costa JF

Practice Guideline. 10.1016/j.nrleng.2021.07.002. 2022


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


Descriptive analysis of the spinal muscular atrophy population treated with Nusinersen included in the CuidAME project

Sotoca Fernandez, J.; (...); Fernandez-Garcia, M.

Meeting Abstract. 10.1016/j.nmd.2024.07.462. 2024

  • Open Access.

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 and Validation of a Clinical Outcome Measure for Adolescents and Adult Patients with Spinal Muscular Atrophy: SMA Life Study Protocol

Rebollo, Pablo; (...); Vazquez-Costa, Juan F.

Article. 10.1007/s40120-023-00571-9. 2024


Design of a Non-Interventional Study to Validate a Set of Patient- and Caregiver-Oriented Measurements to Assess Health Outcomes in Spinal Muscular Atrophy (SMA-TOOL Study).

Madruga-Garrido M; (...); Maurino J

Article. 10.1007/s40120-020-00229-w. 2021


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


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


Distal hereditary motor neuropathies: mutation spectrum and genotype-phenotype correlation.

Frasquet, Marina; (...); Lupo, Vincenzo

Article. 10.1111/ene.14700. 2021


Do we really need to calculate a minimal important difference for ALSFRS-R?: A letter in response to 'Clinically meaningful change: evaluation of the Rasch-built Overall Amyotrophic Lateral Sclerosis Disability Scale (ROADS) and the ALSFRS-R' published in Vol. 24(3-4), pp. 311-316

Vázquez-Costa JF

Article. 10.1080/21678421.2023.2248199. 2024


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


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


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


Estimation of the prevalence and incidence of motor neuron diseases in two Spanish regions: Catalonia and Valencia.

Barceló MA; (...); Saez M

Article. 10.1038/s41598-021-85395-z. 2021


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.

Sivera R; (...); Sevilla T

Article. 10.1212/WNL.0000000000209174. 2024


Expanding the molecular characterization of the CMT2Z disease associated gene MORC2

Sancho, P; (...); Espinos, C

Meeting Abstract. 2019


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 New Cases, Cognitive Changes, and Pathophysiology

de Boer, EMJ; (...); van Es, MA

Review. 10.1212/CPJ.0000000000000834. 2021


Fatigue, pain, breathing, voice, fatigability, sleep, rest and vulnerability as meaningful outcomes in SMA care: the patients ' and caregivers' voice

Povedano, M.; (...); Rebollo, P.

Meeting Abstract. 10.1016/j.nmd.2022.07.209. 2022


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


Guide to Good Humanization Practices in Patients with Rare Diseases. who vadis

Poveda Andres, Jose Luis

Editorial Material. 2022


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

  • Open Access.

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


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


Imaging Biomarkers in Amyotrophic Lateral Sclerosis

Cerda Alberich, Leonor; (...); Marti-Bonmati, Luis

Editorial Material. 10.1007/978-1-0716-1712-0_20. 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 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


Improving Care and Empowering Adults Living with SMA: A Call to Action in the New Treatment Era.

Walter MC; (...); Servais L

Article. 10.3233/JND-200611. 2021


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


Insights into phenotypic variability caused by GARS1 pathogenic variants.

Jiménez-Jiménez J; (...); Sivera R

Article. 10.1111/ene.16416. 2024


Intermediate Repeat Expansion in the ATXN2 Gene as a Risk Factor in the ALS and FTD Spanish Population.

Borrego-Hernandez, Daniel; (...); Garcia-Redondo, Alberto

Article. 10.3390/biomedicines12020356. 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


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

  • Open Access.

La reserva cognitiva como factor modulador del impacto de la cirugía sobre la memoria visual y la denominación en pacientes con epilepsia del lóbulo temporal.

Cano-Lopez, I; (...); Gonzalez-Bono, E

Article. 10.33588/rn.7308.2021109. 2021


Lifesaving Treatments for Spinal Muscular Atrophy: Global Access and Availability

Armengol, Victor D.; (...); Roy, Bhaskar

Meeting Abstract. 2023


Life-Saving Treatments for Spinal Muscular Atrophy: Global Access and Availability.

Armengol, Victor D; (...); Roy, Bhaskar

Article. 10.1212/CPJ.0000000000200224. 2024


Management of flexible cavovarus foot in patients with Charcot-Marie-Tooth disease: midterm results.

Megías-Vericat JE; (...); Poveda-Andrés JL

Article. 2021

  • Open Access.

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


Measuring Outcomes in Adults with Spinal Muscular Atrophy - Challenges and Future Directions - Meeting Report.

Sansone VA; (...); Hagenacker T

Article. 10.3233/JND-200534. 2020


MEASURING THE EXPERIENCE OF LIVING WITH SPINAL MUSCULAR ATROPHY: THE ROLE OF THE SMA INDEPENDENCE SCALE

Vazquez, J. F.; (...); Madruga, M.

Meeting Abstract. 2022


Metabolic Phenotyping in Prostate Cancer Using Multi-Omics Approaches.

Gomez-Cebrian, Nuria; (...); Puchades-Carrasco, Leonor

Article. 10.3390/cancers14030596. 2022


Metyrapone as treatment in the neonatal McCune-Albright syndrome

de Mingo, Carmen; (...); Garcia-Robles, Ana

Article. 10.1515/jpem-2020-0036. 2020


Minimal detectable change and minimal clinically important difference in spinal muscular atrophy patients.

Vázquez-Costa JF, Hervás D

Letter. 10.1111/ene.14780. 2021


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


Moral reasoning and moral conflict in patients of the amyotrophic lateral sclerosis - Frontotemporal dementia spectrum.

Serrano-Pastor, Lucas; (...); Vazquez-Costa, Juan F

Article. 10.1080/17470919.2020.1850521. 2020


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


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 data in adults with SMA

Vazquez-Costa, JF

Letter. 10.1016/S1474-4422(20)30183-6. 2020


NATURAL HISTORY STUDY OF SORD NEUROPATHY

Cortese, Andrea; (...); Zuchner, Stephan

Meeting Abstract. 2022


Neurodegeneration Biomarkers in Adult Spinal Muscular Atrophy (SMA) Patients Treated with Nusinersen

Andres-Benito, Pol; (...); Povedano, Monica

Article. 10.3390/ijms25073810. 2024


New challenges of advanced therapies.

Megías-Vericat JE; (...); Poveda Andrés JL

Article. 10.1016/j.farma.2023.11.008. 2024


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

  • Open Access.

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


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

  • Open Access.

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


PK-GUIDED SWITCH BETWEEN STANDARD HALF-LIFE AND EXTENDED HALF-LIFE FACTOR VIII PRODUCTS

Vericat, JEM; (...); Iorio, A

Meeting Abstract. 2020


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

  • Open Access.

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


Quantitative neuromuscular ultrasound analysis as biomarkers in amyotrophic lateral sclerosis

Rios-Diaz, J; (...); Martinez-Paya, JJ

Article. 10.1007/s00330-018-5943-8. 2019


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 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


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


Spinal Muscular Atrophy Update in Best Practices: Recommendations for Diagnosis Considerations.

Schroth, Mary; (...); Vazquez-Costa, Juan F.

Article. 10.1212/CPJ.0000000000200310. 2024


STATISTICAL SOLUTIONS FOR ANALYTIC CHALLENGES IN REGISTRIES FOR HTA-PURPOSES

Fekete, I; (...); Volmer, T

Meeting Abstract. 2020


Systematic Review of Pharmacogenetics of ABC and SLC Transporter Genes in Acute Myeloid Leukemia

Megías-Vericat JE; (...); Montesinos P

Review. 10.3390/pharmaceutics14040878. 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.nrl.2022.01.008. 2024


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 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

  • Open Access.

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


Treatment of patients with spinal muscular atrophy 5q: Towards a new protocol.

Gomez-Andres, D., Martinez-Moreno, M., Vazquez-Costa, J. F.

Letter. 10.1016/j.nrl.2020.10.003. 2021


Treatment of patients with spinal muscular atrophy 5q: towards a new protocol.

Gómez-Andrés D, Martínez-Moreno M, Vázquez-Costa JF

Letter. 10.1016/j.nrleng.2020.10.005. 2021

  • Open Access.

Treatment of spinal muscular atrophy in European countries: A call to action

Vazquez-Costa, Juan F.; (...); Cattinari, Maria Grazia

Article. 10.1111/ene.15522. 2022


Ultrasound-guided lumbar puncture for nusinersen administration in spinal muscular atrophy patients.

Veiga-Canuto D; (...); Aparici F

Article. 10.1111/ene.14586. 2021


Undetectable levels of adalimumab in clinical practice: Should we say goodbye to the drug?

Bejar, S; (...); Mateu, PN

Meeting Abstract. 2020


USE OF CAPLACIZUMAB IN THE ACQUIRED THROMBOTIC THROMBOCYTOPENIC PURPURA

Dominguez Hernandez, L.; (...); Gomez Segui, I

Meeting Abstract. 2019

  • Open Access.

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 a Set of Instruments to Assess Patient- and Caregiver-Oriented Measurements in Spinal Muscular Atrophy: Results of the SMA-TOOL Study.

Vazquez-Costa, Juan F.; (...); Madruga-Garrido, Marcos

Article. 10.1007/s40120-022-00411-2. 2022


Validation of Neuromyotype: A smart keyboard for the evaluation of spinal muscular atrophy patients

Lizandra Cortes, P.; (...); Vazquez-Costa, J. F.

Article. 10.1016/j.nrl.2022.05.004. 2024


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


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


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


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