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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Bibliografische gegevens
Hoofdauteurs: 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.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: 2007
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Article
Online toegang: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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