#CureLGMDR1

C3 awards grant to University of Florida researchers to investigate calcium handling in LGMD2A/R1

Coalition to Cure Calpain 3 (C3) is driving LGMD2A/R1 research by awarding a new grant to Dr. Elisabeth Barton and Dr. Lan Wei-LaPierre of the University of Florida. The project is titled “Strategies to improve calcium handling in LGMD2A/R1.” LGMD2A/R1

Join Coalition to Cure Calpain 3 in celebrating Rare Disease Day!

Rare Disease Day takes place on the last day of February every year, with the goal of raising awareness about rare diseases and their impact on patients’ lives. Over 300 million people are living with one or more of over

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

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