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
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
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,
Dr. Stephanie Hunn awarded C3 Travel Grant
Coalition to Cure Calpain 3 (C3) is pleased to announce that Stephanie Hunn DPT was awarded a Travel Grant for the presentation of her research at the Annual Congress of the World Muscle Society in Charleston, South Carolina, from October
Coalition to Cure Calpain 3 in partnership with the National Organization for
Rare Disorders (NORD®) launches LGMD2A/Calpainopathy Registry
Today, Coalition to Cure Calpain 3 (C3) in partnership with the National Organization for Rare Disorders (NORD®) launched a study with global reach to research Calpainopathy (including limb-girdle muscular dystrophy type 2A/LGMDR1 and limb-girdle muscular dystrophy type 1I/LGMDD4), a rare
Vita Therapeutics enters licensing agreement with MilliporeSigma
Today, Vita Therapeutics announced they entered a licensing agreement with MilliporeSigma to advance their cell therapy program to treat LGMD2A/R1. This agreement allows Vita to utilize MilliporeSigma’s CRISPR gene editing patents to insert a functional copy of the CAPN3 gene
New “Voice of the Patient” report highlights unmet medical needs of six subtypes of LGMD, including LGMD2A/R1
Today, Coalition to Cure Calpain 3 joins five other LGMD patient organizations (collectively referred to as The LGMD Coalition*) to release the ‘Voice of the Patient Report’ summarizing outcomes from the September 2022 Externally-Led Patient Focused Drug Development Meeting (EL-PFDD).
C3 funds retrospective analysis of clinical outcome assessments
Coalition to Cure Calpain 3 (C3) is pleased to announce the funding of a new research award to Dr. Meredith James and Professor Jordi Díaz-Manera of the John Walter Muscular Dystrophy Research Centre (JWMDRC) at Newcastle University. The project, titled