Edgewise Therapeutics Initiated DUNE Phase 2 Trial of EDG-5506 in Adults with LGMD2I, BMD and McArdle Disease. The Company initiated the DUNE Phase 2 exercise challenge study, to evaluate the effect of EDG-5506 on biomarkers of muscle damage following exercise in adults with LGMD2I, BMD or McArdle disease at a single site in Denmark. The placebo-controlled study is expected to enroll 36 participants for 16 weeks, then continue to an open label extension to 52 weeks. https://lnkd.in/gfigS8Q
New ICD-10 codes for LGMDs! CureLGMD2i is thankful to collaborate with an amazing team of patient organizations and medical experts, making a huge stride for our LGMD community! See the Press Release from MDA
BridgeBio and ML Bio Solutions Announce Positive Phase 2 Data for Limb-girdle Muscular Dystrophy Type 2i (LGMD2i):Press Release
Atamyo Therapeutics announces regulatory approval for their gene therapy trial for LGMD2i/R9!
PTC Therapeutics has decided to discontinue A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I). This study was designed to evaluate the safety and efficacy of deflazacort in participants with LGMD2I. Patients currently enrolled in the Emflaza® study that would like to remain on drug are encouraged to work with their physician to pursue access to treatment for up to 6 months.
