Orphan drug development in muscular dystrophy: Update on two large clinical trials of dystrophin rescue therapies

Authors

Eric P. Hoffman, George Washington UniversityFollow
Edward M. Connor, Children's National Medical Center

Document Type

Journal Article

Publication Date

11-2013

Journal

Discovery Medicine

Volume

Volume 16, Issue 89

Inclusive Pages

233-239

Keywords

Dystrophin--metabolism; Muscular Dystrophies--drug therapy; Muscular Dystrophies--metabolism; Orphan Drug Production

Comments

This is an open access article. Click on link for full-text access.

APA Citation

Hoffman, E.P., Connor, E.M. (2013). Orphan drug development in muscular dystrophy: Update on two large clinical trials of dystrophin rescue therapies. Discovery Medicine, 16(89), 233-239.

Peer Reviewed

1

Open Access

1