Coalition to Cure Calpain 3

New natural history study recruiting individuals with LGMD2A/R1 in France

Coalition to Cure Calpain 3 is pleased to share information regarding a natural history study being conducted in France. Patients must be affiliated with French social security to participate. Additional inclusion criteria is detailed in the flyer below. If you

Vita Therapeutics letter to the LGMD2A/R1 community

Today, Vita Therapeutics published an open letter to the LGMD2A/R1 community announcing some important changes to their development plans.

WATCH: Sarepta presents webinar about new natural history study for individuals living with LGMD2A/R1

Sarepta Therapeutics recently joined C3 to present a community webinar titled “Journey Update for the LGMD2A/R1 Calpainopathy Community.” During the webinar, Sarepta discussed the essential role for natural history studies throughout the drug development process. They then shared the design

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

New LGMD2A/R1 mouse model available at Jackson Labs

Coalition to Cure Calpain 3 (C3) is pleased to share that a new Calpain 3-null mouse strain is available through the Jackson Labs repository. This line is crossed onto an NSG background, resulting in an immunodeficient strain. The NSG.Capn3 KO mice may