Gene Transfer Therapy for Treating Children and Adults With Limb Girdle Muscular Dystrophy Type 2D (LGMD2D)
This study has been completed.
Nationwide Children's Hospital
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Muscular Dystrophy Association
Information provided by (Responsible Party):
Jerry R. Mendell, Nationwide Children's Hospital
First received: June 27, 2007
Last updated: February 4, 2013
Last verified: February 2013
No Study Results Posted on ClinicalTrials.gov for this Study
|Study Status:||This study has been completed.|
|Study Completion Date:||August 2011|
|Primary Completion Date:||June 2011 (Final data collection date for primary outcome measure)|
Mendell JR, Rodino-Klapac LR, Rosales XQ, Coley BD, Galloway G, Lewis S, Malik V, Shilling C, Byrne BJ, Conlon T, Campbell KJ, Bremer WG, Taylor LE, Flanigan KM, Gastier-Foster JM, Astbury C, Kota J, Sahenk Z, Walker CM, Clark KR. Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D. Ann Neurol. 2010 Nov;68(5):629-38. doi: 10.1002/ana.22251.
Mendell JR, Rodino-Klapac LR, Rosales-Quintero X, Kota J, Coley BD, Galloway G, Craenen JM, Lewis S, Malik V, Shilling C, Byrne BJ, Conlon T, Campbell KJ, Bremer WG, Viollet L, Walker CM, Sahenk Z, Clark KR. Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins. Ann Neurol. 2009 Sep;66(3):290-7. doi: 10.1002/ana.21732.