#CureCalpain3

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

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 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

Published Research Alert: Atypical calcium handling in muscles of mouse model of LGMD2A/R1

Limb-Girdle Muscular Dystrophy 2A/R1 (LGMD2A/R1) is caused by changes in a gene called CAPN3, which provides instructions for making a protein called Calpain 3. This protein is specific to skeletal muscle and, among other functions, helps regulate calcium levels inside

Sarepta Therapeutics recruits for clinical outcomes assessment study

C3 is pleased to share this opportunity on behalf of Sarepta Therapeutics: Sarepta welcomes individuals with LGMD2A/R1 to join a clinical outcomes assessment study. To learn more about the study, visit journeyLGMD.com

Volker Straub appointed to C3 Scientific Advisory Board

Coalition to Cure Calpain 3 (C3) is pleased to share that Dr. Volker Straub has been appointed to our Scientific Advisory Board (SAB), joining Dr. Melissa Spencer (Chair), Dr. Kevin Campbell, Dr. Eric Hoffman, and Dr. Louis Kunkel. Professor Volker

C3 hosts Sarepta to present webinar about the JOURNEY natural history study

Click here to register!

C3 attends the 2024 MDA Clinical and Scientific Conference

The Muscular Dystrophy Association held their 2024 Clinical and Scientific Conference on March 3-6 in Orlando, Florida. C3 Scientific Director Dr. Jennifer Levy attended the meeting and shares the following highlights: The MDA Public Policy and Advocacy Team convened representatives

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