A new paper, “Motor Function in LGMD2A/R1: Validation of Clinical Outcome Assessments for Clinical Care and Trial Readiness,” was recently published in the journal Neurology: Genetics. This important, C3-funded initiative is a testament to international cooperation, bringing together leading LGMD
What’s new in LGMD2A/R1 research? Posters highlight new research at the International LGMD Conference
The 2025 International LGMD Conference, hosted by the Speak Foundation, was attended by over 500 LGMD patients and family members, clinicians, and scientists. For the first time, the conference included a scientific poster session for researchers to present their basic, translational, or
C3 Attends the 2025 International LGMD Conference
C3 was proud to be a part of the International LGMD Conference which convened July 18-20 in Orlando, Florida. This event is hosted by the Speak Foundation and focuses on all subtypes of LGMD. Among the highlights: A recording of
LGMD2A/R1 Standards of Care Workshop
Coalition to Cure Calpain 3 (C3) is proud to have partnered with the John Walton Muscular Dystrophy Research Centre, Sarepta Therapeutics, the Speak Foundation, and LGMD Awareness Foundation to sponsor the LGMD2A/R1 Standards of Care (SOC) Workshop. This meeting, organized
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.
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