Portal de recerca
A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION).
Dades bàsiques
- Protocol:
- SRP-9001-303
- EURDRACT:
- 2020-002372-13
- NCT:
- Centre:
- HOSPITAL UNIVERSITARI I POLITÈCNIC LA FE
- Any inici:
- 2023
- Any de finalització:
Documents
- No hi ha documents
Participants
Grups d'Investigació
Finançadors - Promotors
Resultats de l'Assaig Clínic
[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
A multi-criteria decision analysis on the value of nintedanib for interstitial lung diseases.
Zozaya, Neboa; (...); Verde, Luis
Article. 10.1017/S0266462322000459. 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
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
Clinical and Genetic Analysis of Patients With TK2 Deficiency.
Ceballos F; (...); Domínguez-González C
Article. 10.1212/NXG.0000000000200138. 2024
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 usefulness of cerebrospinal fluid biomarkers in Alzheimer Disease.
Pena-Bautista, Carmen; (...); Chafer-Pericas, Consuelo
Article. 10.1111/eci.13910. 2022
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
Correction: Vitamin C and folate status in hereditary fructose intolerance.
Cano A; (...); de Las Heras J
Correction. 10.1038/s41430-023-01334-3. 2023
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
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
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
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
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
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
DRP2 mutations as a cause of Charcot Marie Tooth in Spain
Sivera, R.; (...); Sevilla, T.
Meeting Abstract. 2023
Endothelial Biomarkers Are Superior to Classic Inflammatory Biomarkers in Community-Acquired Pneumonia.
González-Jiménez P; (...); Méndez R
Article. 10.3390/biomedicines12102413. 2024
Endothelial Damage, Neutrophil Extracellular Traps and Platelet Activation in COVID-19 vs. Community-Acquired Pneumonia: A Case-Control Study.
González-Jiménez P; (...); Menéndez R
Article. 10.3390/ijms241713194. 2023
Evaluating the complement C1q levels in serum and cerebrospinal fluid in multiple sclerosis patients: Could it serve as a valuable marker in clinical practice?
Tortosa-Carreres J; (...); Pérez-Miralles FC
Article. 10.1016/j.jneuroim.2024.578428. 2024
Expanding the Clinical Spectrum of DRP2-Associated Charcot-Marie-Tooth Disease.
Article. 10.1212/WNL.0000000000209174. 2024
Extracellular vesicles from dental pulp mesenchymal stem cells modulate macrophage phenotype during acute and chronic cardiac inflammation in athymic nude rats with myocardial infarction.
Amaro-Prellezo, Elena; (...); Sepulveda, Pilar
Article. 10.1186/s41232-024-00340-7. 2024
Genetic Variants Associated with Biological Treatment Response in Inflammatory Bowel Disease: A Systematic Review.
Plaza J; (...); Moret-Tatay I
Article. 10.3390/ijms25073717. 2024
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
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
How can artificial intelligence optimize value-based contracting?
Poveda, Jose Luis; (...); Medrano, Ignacio H.
Letter. 10.1186/s40545-022-00475-3. 2022
Insights into phenotypic variability caused by GARS1 pathogenic variants.
Jiménez-Jiménez J; (...); Sivera R
Article. 10.1111/ene.16416. 2024
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
Life-Saving Treatments for Spinal Muscular Atrophy: Global Access and Availability.
Armengol, Victor D; (...); Roy, Bhaskar
Article. 10.1212/CPJ.0000000000200224. 2024
Long-term reoperation in Tetralogy of Fallot: is it possible to preserve the native pulmonary valve again?
Martineza, Felix Serrano; (...); Molinab, Esteban Peiro
Article. 10.1016/j.circv.2022.01.009. 2022
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
Modeling Cardiotoxicity in Pediatric Oncology Patients Using Patient-Specific iPSC-Derived Cardiomyocytes Reveals Downregulation of Cardioprotective microRNAs.
Reinal, Ignacio; (...); Sepulveda, Pilar
Article. 10.3390/antiox12071378. 2023
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
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
Neutrophil Extracellular Traps and Platelet Activation for Identifying Severe Episodes and Clinical Trajectories in COVID-19
González-Jiménez P; (...); Menéndez R
Article. 10.3390/ijms24076690. 2023
New challenges of advanced therapies.
Megías-Vericat JE; (...); Poveda Andrés JL
Article. 10.1016/j.farma.2023.11.008. 2024
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
Outcomes of a Pilot Newborn Screening Program for Spinal Muscular Atrophy in the Valencian Community.
Berzal-Serrano, Alba; (...); Millan, Jose M
Article. 10.3390/ijns11010007. 2025
Pain in Children and Adolescents with Spinal Muscular Atrophy: A Longitudinal Study from a Patient Registry.
Pitarch-Castellano I; (...); Madruga-Garrido M
Article. 10.3390/children10121880. 2023
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
Pilot study for the implementation of newborn screening for spinal muscular atrophy in Valencia
Berzal-Serrano, Alba; (...); Millan, Jose M.
Meeting Abstract. 10.2174/1871530323666230914122955. 2024
Predictive potential of serum and cerebrospinal fluid biomarkers for disease activity in treated multiple sclerosis patients.
Tortosa-Carreres J; (...); Pérez-Miralles FC
Article. 10.1016/j.msard.2024.105734. 2024
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
Prognostic value of cerebrospinal fluid biomarkers in mild cognitive impairment due to Alzheimer disease.
Lopez-Cuevas, R; (...); Chafer-Pericas, C
Article. 10.1016/j.nrleng.2020.07.024. 2023
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
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
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 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
Spinocerebellar Ataxia 36 is a Frequent Cause of Hereditary Ataxia in Eastern Spain
Baviera-Munoz, Raquel; (...); Bataller, Luis
Article. 10.1002/mdc3.13740. 2023
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 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
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
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