New data from the GRASP-001 study identify clinical outcome assessments for future LGMD2A/R1 clinical trials A team of LGMD researchers, called the GRASP-LGMD Consortium, published the baseline results of their LGMD2A/R1 clinical outcome study in the Annals of Clinical and Translational Neurology.
Upcoming Event: International LGMD Conference
Don’t miss out on the upcoming International LGMD Conference! Space is limited so register now: https://nationallimbgirdlemusculardystrophyconference.com/ And be sure to join C3 at our complimentary reception! Email us directly at info@curecalpain3.org to reserve your spot by June 30.
C3 Celebrates Rare Disease Day
Sarepta Therapeutics Update
Sarepta Therapeutics hosted an Earnings Call on February 26, 2025 where they announced that they expect to file an Investigational New Drug Application (IND) for their LGMD2A/R1 gene therapy program in 2025. An IND is submitted to the FDA
2024 Marked by Collaboration, Community, Commitment
As 2024 comes to a close, Coalition to Cure Calpain 3 (C3) is energized as we look back on a year marked by collaboration, community, and a continued commitment to drive research toward a cure for individuals living with limb-girdle
Coalition to Cure Calpain 3 and Muscular Dystrophy Association partner to fund gene therapy grant
Muscular Dystrophy Association (MDA) and Coalition to Cure Calpain 3 (C3) partner to fund $300,000 research grant for gene therapy development in limb-girdle muscular dystrophy. This grant supports the work of Melissa Spencer, PhD, Director of the Neuromuscular Division in
C3 Scientific Meeting Recap
Coalition to Cure Calpain 3 (C3) recognizes that collaboration and data sharing are integral to drug discovery and therapy development. To foster these interactions, we hosted an in-person meeting focused on advances in clinical trial readiness and treatment development for
C3 ANNOUNCES REQUEST FOR APPLICATIONS FOR RESEARCH INTO CALPAINOPATHY
SYNOPSIS Coalition to Cure Calpain 3 (C3) provides funding for research and translational projects to expand the understanding of calpain 3 and Calpainopathy (including LGMD2A/R1 and LGMD1I/D4) to enable development of therapeutic approaches for this disease. This award is intended
C3 joins Task Force to advance therapeutic development for limb-girdle muscular dystrophies
The Coalition to Cure Calpain team is looking forward to working with the Critical Path Institute (C-Path), ML Bio Solutions, and our LGMD organization colleagues on this task force “dedicated to advancing therapeutic development for limb-girdle muscular dystrophies (LGMDs).” Read
C3 celebrates 10th annual LGMD Awareness Day!
There is great strength in awareness, understanding, and unity. The LGMD community demonstrates global connectivity each year when we campaign together and gain recognition for LGMD. With over 30 different genetic types of LGMD, the act of collaborating globally makes