Research Projects

Sarepta announces gene therapy program for LGMD2A/R1

Today, Sarepta Therapeutics, Inc. announced that it executed an exclusive license agreement for an LGMD2A/R1 gene therapy program developed by Dr. Zarife Sahenk at Nationwide Children’s Hospital. Preclinical research conducted by Dr. Sahenk provided early proof of concept data for

FUNDING OPPORTUNITY! Coalition to Cure Calpain 3 Issues Request for Applications for Research Related to LGMD2A/R1

Coalition to Cure Calpain 3 requests applications for research and translational projects related to calpain 3 and limb-girdle muscular dystrophy type 2A (LGMD2A, also called LGMDR1 Calpain 3-related). The goal of the RFA is to enable the development of therapeutic

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

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

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 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

C3 Celebrates a Decade of Achievement

Coalition to Cure Calpain 3 (C3) has come a long way in ten years in driving science and awareness to treat limb girdle muscular dystrophy, type 2A (known as LGMD2A/R1, LGMD R1 Calpain 3 related, or calpainopathy). In celebration of ten

Patient Recruitment Underway for C3-Sponsored Natural History Study

The GRASP-LGMD 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 Virginia

Notes from 23rd annual meeting of the American Society for Gene and Cell Therapy

The American Society for Gene and Cell Therapy (ASGCT) annual meeting kicked off on Tuesday, May 12, 2020. Due to COVID-19, the meeting was held virtually. This meeting covers the field’s latest and most innovative science across all diseases and