C3 News

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

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

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

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).