Author: Jennifer Levy

C3 Awards Research Grant to Drs. Melissa Spencer and Jeffrey Chamberlain to Develop Gene Therapy Vectors for LGMD2A/R1

Coalition to Cure Calpain 3 (C3) is delighted to announce a newly-funded research grant to develop a gene therapy for LGMD2A/R1 using vectors optimized for gene delivery to muscles most affected by the disease. This work involves a multi-lab collaboration

C3 Selected as Platinum Member of the National Organization for Rare Diseases

Coalition to Cure Calpain 3 (C3) has joined the National Organization for Rare Diseases (NORD) as a Platinum Member. This level of membership represents the highest level of integrity and transparency for patient advocacy organizations involved in medical research, drug

LGMD2A/R1 Participants Sought for COVID-19 Survey

Coalition to Cure Calpain 3 is sharing the following communication from the University of Rochester regarding a voluntary research survey to be completed by those living with LGMD2A/R1, a form of calpainopathy, or by their caregivers. Study Investigator: Rabi Tawil, MD

LGMD2A/R1 Patient Enrollment Underway for C3-Sponsored Natural History Study

The GRASP-LGMD consortium is currently recruiting for a natural history study for individuals with mutations in CAPN3 (calpain 3). This study will inform the design of future therapeutic trials and a better understanding of the disease. Participants can now enroll at

Organizations team up to urge health officials to prioritize the neuromuscular disease community for COVID-19 vaccine access

As distribution of COVID-19 vaccines begins, the Muscular Dystrophy Association led Coalition to Cure Calpain 3 and 18 other partner organizations in advocating to over 55 states and localities, urging health officials to prioritize access for the neuromuscular disease community.

C3 is Poised to Make a Difference in the Next Ten Years and Beyond

Coalition to Cure Calpain 3 (C3) has made tremendous progress in driving research towards a cure for limb-girdle muscular dystrophy type 2A (LGMD2A/R1) and raising global disease awareness. Over the last 10 weeks of highlights, you have read all the WAYS

C3 is Driving Progress Towards a Cutting-Edge Cure

Coalition to Cure Calpain 3 (C3) is bringing cutting-edge science to drive progress toward a cure for limb-girdle muscular dystrophy type 2A (LGMD2A/R1). Some of our proudest work yet has been to help support and harness incredible new scientific understanding

C3 is Expanding the Field of LGMD2A/R1 Researchers

Coalition to Cure Calpain 3 (C3) is attracting more researchers to study – and ultimately cure – limb-girdle muscular dystrophy 2A (LGMD2A/R1). We have worked tirelessly to bring more scientists, research, and dollars to focus on and unravel our understudied

FDA Patient Listening Session on LGMDs held October 20, 2020

On October 20th, 2020, the LGMD community was able to meet with the FDA to share our patient experiences. This was a patient-led listening session organized by a consortium of advocacy organizations, including Coalition to Cure Calpain 3. The session

C3 is Increasing Scientific Collaboration to Fight LGMD2A/R1

Coalition to Cure Calpain 3 (C3) initiates scientific collaboration to bring the best minds together to fight limb-girdle muscular dystrophy type 2A (LGMD2A/R1). Working to cure LGMD2A/R1 requires connecting ideas, scientists, clinicians, patient groups and companies to elevate research to