Attention Individuals with LGMD2A/R1: Coalition to Cure Calpain 3 wants to hear from you! If you have participated in clinical research, such as a natural history study, an observational study, or a clinical trial, please follow this link to answer
What’s on the Horizon for LGMD2A/R1?
Dr. Jennifer Levy, Coalition to Cure Calpain 3 Scientific Director, is a proud contributor to the Winter 2022 issue of LGMD News. This magazine, a project of The Speak Foundation, publishes up-to-date information for the LGMD community. Dr. Levy’s article
C3 to Participate in Externally-Led PFDD Meeting with FDA
If you are living with LGMD2A/R1, you will have the opportunity to participate in an externally-led Patient-Focused Drug Development Meeting (EL-PFDD) with the FDA on September 23, 2022. C3 is proud to be one of six LGMD organizations to collaborate
A Special Message from Lionel Messi to the C3 Community
C3 is thrilled to share a special video message from soccer great Lionel Messi to promote September 30th as Limb-Girdle Muscular Dystrophy Awareness Day! CLICK HERE to donate to C3 to drive research for a cure, or text CALPAIN to
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