Coalition to Cure Calpain 3

Community Health Clinic publishes newsletter focused on LGMDs

LGMD2A/R1 is considered a rare disease because it affects only about 1 in 100,000 people. However, certain areas of the world have a higher prevalence. Indiana’s Adams and Jay Counties have the highest concentration of individuals living with LGMD2A/R1: more

New Research Alert: GRASP-001 Study

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.

Sarepta Community Update on LGMD Programs

Sarepta Therapeutics recently published a Community Letter to share updates on their limb-girdle muscular dystrophy gene therapy programs. Click here to access the letter on Sarepta’s website. Sarepta is enrolling participants in their LGMD2A/R1 natural history study cohort while simultaneously

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

Upcoming webinar: The Power of Data Sharing and Collaboration Across LGMDs in a Pre-Competitive, Neutral Environment

Thursday, December 12 at 11 AM EST This event will feature Dr. Volker Straub, a renowned neuromuscular genetics expert, Director of the John Walton Muscular Dystrophy Research Centre at Newcastle University, and member of C3’s Scientific Advisory Board. Dr. Straub will

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

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