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The SMA Clinical Trial Readiness Program: creation and evaluation of a program to enhance SMA trial readiness in the United States

Spinal muscular atrophy (SMA) is a rare neuromuscular disease with a rapidly evolving treatment landscape. To better meet the needs of trial sponsors and the patient community in the United States (US) in this evolving context, Cure SMA established a clinical trial readiness program for new and pros...

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Detalles Bibliográficos
Autores principales:	Peterson, Ilse, Cruz, Rosángel, Sarr, Fatou, Stanley, Ann Marie, Jarecki, Jill
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2020
Materias:	Position Statement
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7564894/ https://www.ncbi.nlm.nih.gov/pubmed/32443972 http://dx.doi.org/10.1186/s13023-020-01387-8

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