Implementing a Global Expanded Access Program (EAP) for Infantile-Onset Spinal Muscular Atrophy (Type I): Understanding the Imperative, Impact and Challenges

Nusinersen is the first disease-modifying therapy approved for the treatment of spinal muscular atrophy (SMA), a rare genetic disorder characterized by severe progressive muscular atrophy and weakness. An expanded access program (EAP) provides investigational treatment to patients without other trea...

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Detalles Bibliográficos
Autores principales: Yong, Jonathan, Moffett, Megan, Lucas, Sam
Formato: Online Artículo Texto
Lenguaje:English
Publicado: IOS Press 2019
Materias:
Short Communication
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6598208/
https://www.ncbi.nlm.nih.gov/pubmed/31127730
http://dx.doi.org/10.3233/JND-190387

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