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 therapy,
C3 is delighted to share that a research grant has been awarded to Dr. Peter Davies of Queen’s University in Ontario, Canada. The project, titled “Solving the structure of Calpain 3 and assessing the impact of LGMD2A/R1,” aims to determine
C3 is pleased to announce the funding of a new research project to Dr. Christopher Heier, of Virginia Commonwealth University in the USA, and Dr. Utkarsh Dang, of Carleton University in Canada. The project is titled “Comparative proteomics of LGMD2A/R1
A C3 Champion has pledged to match every dollar raised – up to $15,000 – between now and December 9. This generous Giving Tuesday donation is made in honor of Matt Straface. Greetings! My name is Matt and it has
A new paper, “Motor Function in LGMD2A/R1: Validation of Clinical Outcome Assessments for Clinical Care and Trial Readiness,” was recently published in the journal Neurology: Genetics. This important, C3-funded initiative is a testament to international cooperation, bringing together leading LGMD
A group of researchers from the Heimer Institute for Muscle Research in Bochum, Germany have characterized a Capn3-deficient mouse strain. This is one of four strains recently generated by Jackson Laboratories as part of a Coalition to Cure Calpain 3
Don’t miss out on the upcoming International LGMD Conference! Space is limited so register now: https://nationallimbgirdlemusculardystrophyconference.com/ And be sure to join C3 at our complimentary reception! Email us directly at info@curecalpain3.org to reserve your spot by June 30.
Sarepta Therapeutics recently published a Community Letter to share updates on their limb-girdle muscular dystrophy gene therapy programs. Click here to access the letter on Sarepta’s website. Sarepta is enrolling participants in their LGMD2A/R1 natural history study cohort while simultaneously
Muscular Dystrophy Association (MDA) and Coalition to Cure Calpain 3 (C3) partner to fund $300,000 research grant for gene therapy development in limb-girdle muscular dystrophy. This grant supports the work of Melissa Spencer, PhD, Director of the Neuromuscular Division in
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