RESTORE: A Prospective Multinational Registry of Patients with Genetically Confirmed Spinal Muscular Atrophy - Rationale and Study Design

BACKGROUND: Dramatic improvements in spinal muscular atrophy (SMA) treatment have changed the prognosis for patients with this disease, leading to important new questions. Gathering representative, real-world data about the long-term efficacy and safety of emerging SMA interventions is essential to...

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Detalles Bibliográficos
Autores principales: Finkel, Richard S., Day, John W., De Vivo, Darryl C., Kirschner, Janbernd, Mercuri, Eugenio, Muntoni, Francesco, Shieh, Perry B., Tizzano, Eduardo, Desguerre, Isabelle, Quijano-Roy, Susana, Saito, Kayoko, Droege, Marcus, Dabbous, Omar, Khan, Farid, Renault, Lydie, Anderson, Frederick A., Servais, Laurent
Formato: Online Artículo Texto
Lenguaje:English
Publicado: IOS Press 2020
Materias:
Research Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7739962/
https://www.ncbi.nlm.nih.gov/pubmed/32039859
http://dx.doi.org/10.3233/JND-190451

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7739962/
https://www.ncbi.nlm.nih.gov/pubmed/32039859
http://dx.doi.org/10.3233/JND-190451

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