#CureCalpain3

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

Trial readiness study for individuals with LGMD2A/R1

Please contact either Ruby Langeslay or Jennifer Raymond at Virginia Commonwealth University for more information

KNOW YOUR CODE: Why the new ICD-10 codes matter, and what you can do to confirm your records

Did you know that new ICD-10 codes were assigned for limb-girdle muscular dystrophies (LGMDs) in October 2022? Patients with LGMD2A/R1, autosomal recessive LGMD due to Calpain 3-dysfunction, have been given a diagnosis code of G71.032. Patients with LGMD1i/D4, autosomal dominant

Countdown to the New Year – 2023 Edition

This year, C3 collaborated with 5 other LGMD organizations to publish the Voice of the Patient Report, summarizing the 2022 Externally-Led Patient Focused Drug Development Meeting and highlighting the unmet medical needs of the LGMD community. READ MORE Connected with

Genetic testing for LGMDs

Limb-girdle muscular dystrophies (LGMDs) were characterized in the clinic before anyone knew what caused the diseases. Consequently, several different diseases that all looked the same to physicians were lumped together into one category that they called “LGMD.” As science advanced,

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