SMA-EUROPE workshop report: opportunities and challenges in developing clinical trials for spinal muscular atrophy in Europe

Spinal muscular atrophy (SMA) is the most common lethal recessive disease in childhood, and there is currently no effective treatment to halt disease progression. The translation of scientific advances into effective therapies is hampered by major roadblocks in clinical trials, including the complex...

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Detalles Bibliográficos
Autores principales: Kayadjanian, Nathalie, Burghes, Arthur, Finkel, Richard S, Mercuri, Eugenio, Rouault, Francoise, Schwersenz, Inge, Talbot, Kevin
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2013
Materias:
Letter to the Editor
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3627630/
https://www.ncbi.nlm.nih.gov/pubmed/23514578
http://dx.doi.org/10.1186/1750-1172-8-44

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3627630/
https://www.ncbi.nlm.nih.gov/pubmed/23514578
http://dx.doi.org/10.1186/1750-1172-8-44

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