A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy.
Mendell JR, Sahenk Z, Malik V, Gomez AM, Flanigan KM, Lowes LP, Alfano LN, Berry K, Meadows E, Lewis S, Braun L, Shontz K, Rouhana M, Clark KR, Rosales XQ, Al-Zaidy S, Govoni A, Rodino-Klapac LR, Hogan MJ, Kaspar BK.
Mendell JR, et al. Among authors: kaspar bk.
Mol Ther. 2015 Jan;23(1):192-201. doi: 10.1038/mt.2014.200. Epub 2014 Oct 17.
Mol Ther. 2015.
PMID: 25322757
Free PMC article.
Clinical Trial.