#CureCalpain3

Published Research Alert: CAPN3 gene therapy improves muscles function in a mouse model of limb-girdle muscular dystrophy type 2A/R1 (LGMD2A/R1)

Coalition to Cure Calpain 3 (C3) is pleased to announce the publication of important research undertaken by Dr. Zarife Sahenk, Dr. Jerry Mendell, and colleagues at Nationwide Children’s Hospital in Columbus, Ohio. The paper, titled “Systemic delivery of AAVrh74.tMCK.hCAPN3 rescues the

New publication illuminates the effects of the COVID-19 pandemic on people with muscular dystrophy

A new publication examines the social and health impacts of the COVID-19 pandemic and the associated social guidelines on people living with muscular dystrophies. The authors developed a COVID-19 Impact Survey for adults living with facioscapulohumeral muscular dystrophy, myotonic dystrophy,

Will YOU help advance LGMD2A/R1 research by participating in the GRASP-LGMD Natural History Study?

Natural history studies collect information about the symptoms and progression of a disease in the absence of an intervention. It is vital to understand natural history so that researchers can plan for clinical trials to determine if an investigational drug

Help us make a big change for the LGMD Community

If you live in the US, we need your help! Coalition to Cure Calpain 3 has been working with Muscular Dystrophy Association (MDA) and other LGMD foundations to develop diagnostic codes specific to LGMD. Currently, anyone diagnosed with LGMD is

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

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