Author: Jennifer Levy

Limb Girdle Muscular Dystrophy Consortium Forms to Prepare for Clinical Trials

Big news for Limb Girdle Muscular Dystrophies (LGMDs)! The Muscular Dystrophy Association (MDA) recently awarded a clinical research network grant to Dr. Nicholas Johnson, vice chair of research and associate professor of neurology at Virginia Commonwealth University, to establish an

C3 Awards Gene Therapy Initiative Grant to Dr. Zarife Sahenk

FOURTH GENE THERAPY INITIATIVE GRANT SUPPORTS PROMISING RESEARCH AT NATIONWIDE CHILDREN’S HOSPITAL Coalition to Cure Calpain 3 (C3) is excited to announce that a new research grant has been awarded to Dr. Zarife Sahenk, Professor of Pediatrics and Neurology at

C3 Statement Regarding Partnership Between Myonexus and Sarepta Therapeutics

Sarepta Therapeutics recently announced a partnership with Myonexus Therapeutics focused on developing gene therapies for five subtypes of LGMD: 2B, 2C, 2D, and 2L. The agreement states that Sarepta will make an upfront payment of $60 million. If development-related milestones

Coalition to Cure Calpain 3 Awards Grant to Dr. Kathryn Wagner

Coalition to Cure Calpain 3 (C3) is pleased to announce that we are funding a new research grant to Dr. Kathryn Wagner. Dr. Wagner, Director of the Center for Genetic Muscle Disorders at the Kennedy Krieger Institute and Professor of

C3 Gene Therapy Initiative Awards Research Grant to Dr. Isabelle Richard

Coalition to Cure Calpain 3 (C3) is excited to announce that a new research grant has been awarded to Dr. Isabelle Richard, Researcher at Généthon, to analyze a novel model of calpainopathy and utilize it for the development of adeno-associated

DID YOU KNOW … there are two ways that calpainopathy can be inherited?

DID YOU KNOW … there are two ways that calpainopathy can be inherited? In over 90% of calpainopathy cases, it is inherited in an autosomal recessive manner. This means that it can be passed on through families, and that siblings

C3 Announces Gene Therapy Initiative

PRIME TIME FOR GENE THERAPY While its potential has been discussed and studied for decades, 2017 was a year that showed the promise of gene therapy becoming a reality for patients. The FDA approved the first gene therapies in the

Do you have genetic confirmation of your LGMD diagnosis?

Limb Girdle Muscular Dystrophies (LGMDs) were characterized in the clinic before anyone knew what caused the diseases. Consequently, a bunch of different diseases that all looked the same to physicians were lumped together into one category that they called “LGMD.”

C3 Introduces New Travel Grant Program for Calpainopathy Researchers

Coalition to Cure Calpain 3 (C3) will award Travel Grants to foster early career investigators who are currently conducting preclinical or clinical research in the area of calpainopathy (also known as Limb Girdle Muscular Dystrophy Type 2A or LGMD2A). The

C3 recognizes September 30 “Limb Girdle Muscular Dystrophy Awareness Day” with research grant

Coalition to Cure Calpain 3 (C3) is proud to support September 30 “Limb Girdle MD Awareness Day” with the announcement that we are awarding a new research grant to Dr. Michele Calos, Professor in the Department of Genetics at Stanford