Ongoing Research & Trials
Clinical Trials:
Gene Therapy -
- Sponsor: Sarepta Therapeutics, Inc.
- Recruitment Status: Closed
Natural History Studies:
Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy (LGMD) (GRASP-01-001)
- Sponsor: Virginia Commonwealth University
- Recruitment Status: Active
- Sponsor: Sarepta Therapeutics, Inc.
- Recruitment Status: Closed
Skeletal Muscle Biomarkers in People With Fragile Sarcolemmal Muscular Dystrophy
- Sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- Recruitment Status: Active, Not Recruiting
Research Highlights
Publications
Gene Therapy
Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion - July 2019
Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D - November 2010
Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins - September 2009
Early adenovirus-mediated gene transfer effectively prevents muscular dystrophy in alpha-sarcoglycan-deficient mice - August 2000
Small Molecule
Dual Blockade of Misfolded Alpha-Sarcoglycan Degradation by Bortezomib and Givinostat Combination - PubMed - April 2022
Alpha-Sarcoglycan Mouse Models
We have multiple alpha-sarcoglycan mouse models available for research:
Mutant Mouse Resource and Research Center (MMRRC) -
Jackson Laboratory -
B6.129S6-Sgcatm2Kcam/J (stock #008275)
B6.Cg-Sgcatm1KamTg(Ckm-SGCE)1Kcam/J (stock #009651)
Genetic Tools
Antibody Resources
Abcam antibodies against alpha-sarcoglycan
- Anti-alpha Sarcoglycan antibody [Ad1/20A6] (ab49451)
- Anti-alpha Sarcoglycan antibody (ab92522)
- Anti-alpha Sarcoglycan antibody (ab130657)
Links on Biocompare to antibodies from 16 different vendors
Links on Antibody Resource to antibodies from 12 different vendors
Additional Resources
Bioz analyzes, aggregates, and structures data from more than 3.6 million articles and many different resources related to alpha-sarcoglycan can be found on this website.
Together for a Cure
Your generous donation will help support research to find effective treatments or a cure for
LGMD2D / R3.
Support LGMD2D Warriors
There are currently no approved treatments or a cure for LGMD2D. The incredible warriors fighting this disease around the world need your support. Please consider making a donation—no dollar is too small, and every dollar goes directly toward raising LGMD2D/R3 awareness, advocating for families, and driving the development of life-changing treatments. Join us in this critical mission. Your support truly makes a difference.