C3 News

Carol Abraham appointed to Patient Engagement Collaborative

Today, the U.S. Food and Drug Administration (FDA), in collaboration with the Clinical Trials Transformation Initiative (CTTI), announced that Carol Abraham has been appointed to the Patient Engagement Collaborative (PEC) and will serve a two year term. Carol is the

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

Together we are STRONGER: 7th Annual LGMD Awareness Day is coming up!

There is great strength in awareness, understanding, and unity. The LGMD community demonstrates global connectivity each year when we campaign together and gain recognition for LGMD. With over 30 different genetic types of LGMD, the act of collaborating globally makes

Register now for the 2021 International LGMD Conference

Are you going to the 2021 International LGMD Conference? It should be an amazing event and C3 is looking forward to participating. C3 Scientific Director Dr. Jennifer Levy will moderate a group discussion and C3 Director of Community Outreach Carol

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