A Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy

Becker muscular dystrophy (BMD) is a variant of dystrophin deficiency resulting from DMD gene mutations. Phenotype is variable with loss of ambulation in late teenage or late mid-life years. There is currently no treatment for this condition. In this BMD proof-of-principle clinical trial, a potent m...

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Foilsithe in:Mol Ther
Main Authors: Mendell, Jerry R, Sahenk, Zarife, Malik, Vinod, Gomez, Ana M, Flanigan, Kevin M, Lowes, Linda P, Alfano, Lindsay N, Berry, Katherine, Meadows, Eric, Lewis, Sarah, Braun, Lyndsey, Shontz, Kim, Rouhana, Maria, Clark, Kelly Reed, Rosales, Xiomara Q, Al-Zaidy, Samiah, Govoni, Alessandra, Rodino-Klapac, Louise R, Hogan, Mark J, Kaspar, Brian K
Formáid: Artigo
Teanga:Inglês
Foilsithe: Nature Publishing Group 2015
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Original Article
Rochtain Ar Líne:https://ncbi.nlm.nih.gov/pmc/articles/PMC4426808/
https://ncbi.nlm.nih.gov/pubmed/25322757
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2014.200
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