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Perspectives on Clinical Trials in Spinal Muscular Atrophy

Spinal muscular atrophy is one of the most heterogeneous of the single-gene neuromuscular disorders. The broad spectrum of severity, with onset from the prenatal period to adulthood, presents unique challenges in the design and implementation of clinical trials. The clinical classification of subjec...

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Detalhes bibliográficos
Main Authors: Swoboda, Kathryn J., Kissel, John T., Crawford, Thomas O., Bromberg, Mark B., Acsadi, Gyula, D'Anjou, Guy, Krosschell, Kristin J., Reyna, Sandra P., Schroth, Mary K., Scott, Charles B., Simard, Louise R.
Formato: Artigo
Idioma:Inglês
Publicado em: 2007
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3260051/
https://ncbi.nlm.nih.gov/pubmed/17761650
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1177/0883073807305665
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