Coalition to Cure Calpain 3 (C3) recognizes that collaboration and data sharing are integral to drug discovery and therapy development. To foster these interactions, we hosted an in-person meeting focused on advances in clinical trial readiness and treatment development for
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
What can YOU do to drive LGMD2A/R1 research forward? Participate in a natural history study!
What is a natural history study? A natural history study is a research study that collects information about the genetics, symptoms, and lived experiences of individuals. These studies are considered observational, because they do not include an interventional drug or
C3 joins Task Force to advance therapeutic development for limb-girdle muscular dystrophies
The Coalition to Cure Calpain team is looking forward to working with the Critical Path Institute (C-Path), ML Bio Solutions, and our LGMD organization colleagues on this task force “dedicated to advancing therapeutic development for limb-girdle muscular dystrophies (LGMDs).” Read
C3 celebrates 10th annual LGMD Awareness Day!
There is great strength in awareness, understanding, and unity. The LGMD community demonstrates global connectivity each year when we campaign together and gain recognition for LGMD. With over 30 different genetic types of LGMD, the act of collaborating globally makes
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
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