Portal de investigación
A Phase 1b, Double-Blinded, Randomized, Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, Immunogenicity, and Efficacy of ARGX-119 in Adult Participants With DOK7 Congenital Myasthenic Syndromes.
Datos básicos
- Protocolo:
- ARGX-119-2302
- EUDRACT:
- NCT:
- Centro:
- HOSPITAL UNIVERSITARI I POLITÈCNIC LA FE
- Año de incio:
- 2024
- Año de finalización:
Documentos
- No hay documentos
Participantes
Grupos
Financiadores - Promotores
Resultados del Ensayo Clínico
[Translated article] Knowledge of biological therapy in patients with immune-mediated diseases. BIOINFO study.
Segui-Solanes, Carlos; (...); Borras-Blasco, Joaquin
Article. 10.1016/j.farma.2024.08.002. 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
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 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
Cost-effectiveness and cost-utility analysis of haemate-P versus other von willebrand disease treatments in Spain.
Megias-Vericat, Juan E; (...); Yan, Songkai
Article. 10.1080/13696998.2025.2474886. 2025
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
Development and testing of a mobile app for systematic collection of patient-reported outcomes (PROs) in patients with hemophilia.
Megias-Vericat, J E; (...); Poveda Andres, J L
Letter. 10.1016/j.thromres.2024.109122. 2024
Insights into phenotypic variability caused by GARS1 pathogenic variants.
Jiménez-Jiménez J; (...); Sivera R
Article. 10.1111/ene.16416. 2024
Mental, emotional and social dimensions of quality of life and their relationship with physical and functional status in adults with haemophilia.
Rodríguez MA; (...); Chimeno-Hernández A
Article. 10.1016/j.thromres.2024.109181. 2024
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
New challenges of advanced therapies.
Megías-Vericat JE; (...); Poveda Andrés JL
Article. 10.1016/j.farma.2023.11.008. 2024
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
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
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
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