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  3. Estudio multicentrico de continuación en 2 partes, aleatorizado, controlado con placebo, doble ciego para investigar la seguridad, tolerabilidad, farmacocinética, farmacodinamica y eficacia de RO7204239 en combinación con ridisplam (RO7034067) en pacientes con atrofia muscular espinal.

Estudio multicentrico de continuación en 2 partes, aleatorizado, controlado con placebo, doble ciego para investigar la seguridad, tolerabilidad, farmacocinética, farmacodinamica y eficacia de RO7204239 en combinación con ridisplam (RO7034067) en pacientes con atrofia muscular espinal.

Datos básicos

Protocolo:
BN42644
EUDRACT:
2021-003417-19
NCT:
Centro:
HOSPITAL UNIVERSITARI I POLITÈCNIC LA FE
Año de incio:
2023
Año de finalización:
ENSAYO CLÍNICO

Documentos

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Participantes

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Grupos

Financiadores - Promotores

F. HOFFMAN-LA ROCHE LTD

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

  • Open Access.

[Translated article] New challenges in advanced therapies.

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

Article. 10.1016/j.farma.2024.05.001. 2024

  • Open Access.

[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


A Gadolinium(III) Complex Based on Pyridoxine Molecule with Single-Ion Magnet and Magnetic Resonance Imaging Properties.

Orts-Arroyo, Marta; (...); Martinez-Lillo, Jose

Article. 10.3390/ijms25042112. 2024

  • Open Access.

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


Benefits of physical activity self-monitoring in patients with haemophilia: a prospective study with one-year follow-up.

Pérez-Alenda S; (...); Núñez-Cortés R

Article. 10.1111/hae.14988. 2024

  • Open Access.

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


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


Lateral-type posterior fossa ependymomas in pediatric population

Miranda-Lloret, Pablo; (...); Llavador, Gemma

Article. 10.1016/j.neucir.2023.10.002. 2024

  • Open Access.

Lateral-type posterior fossa ependymomas in pediatric population.

Miranda-Lloret, Pablo; (...); Llavador, Gemma

Article. 10.1016/j.neucie.2023.10.001. 2024


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

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

Article. 10.1212/CPJ.0000000000200224. 2024

  • Open Access.

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

  • Open Access.

New challenges of advanced therapies.

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

Article. 10.1016/j.farma.2023.11.008. 2024

  • Open Access.

Onasemnogene Abeparvovec Administration via Peripherally Inserted Central Catheter: A Case Report.

Pitarch Castellano, Inmaculada; (...); Poveda Andres, Jose L

Case Reports. 10.3390/children11050590. 2024

  • Open Access.

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

  • Open Access.

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.

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

  • Open Access.

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


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