C3 SUPPORTS RESEARCH GROUP FOR NEUROMUSCULAR DISEASES Coalition to Cure Calpain 3 (C3) is pleased to announce that a research grant has been awarded to Dr. Jaakko Sarparanta, researcher at the Research Group for Neuromuscular Diseases led by Dr. Bjarne Udd at Folkhälsan Research Center and Department of Medical Genetics, University of Helsinki, Finland. The project, titled “CAPN3-mediated proteolytic processing of…
Read MoreNew report confirms calpainopathy is most common type of LGMD
A paper recently published by Madhuri Hegde, Emory University, and colleagues analyzed the outcomes of genetically sequencing a large group of patients with limb girdle weakness in the United States. This study is unique in that it included a very large cohort of patients – 4656 – who were enrolled in the Muscular Dystrophy Association…
Read MoreC3 Scientific Conference 2018
On September 12 – 13, 2018, Coalition to Cure Calpain 3 hosted the “C3 Scientific Conference” in Arlington, Virginia. In attendance were prominent neuromuscular clinicians, world renowned research scientists from academia and industry, and representatives from various funding agencies. This included C3’s Scientific Advisory Board and C3 grant recipients. The goal of the conference was…
Read MoreC3 Presents at World Muscle Society Congress
Coalition to Cure Calpain 3 (C3) attended the 23rd International Annual Congress of the World Muscle Society, October 2-6 2018 in Mendoza, Argentina. Dr. Jennifer Levy, C3 Scientific Director, represented C3 and presented a poster detailing our mission, registry, and research grants program. Attendees include neuromuscular clinicians, academics, research scientists, and industry leaders from around the…
Read MoreCoalition to Cure Calpain 3 Provides Gene Therapy Initiative Updates
Two new research projects funded to explore potential treatments for calpainopathy/LGMD2A $500,000+ committed to gene therapy research since initiative launched in 2017 Coalition to Cure Calpain 3 (C3), a non-profit patient advocacy organization focused on identifying and advancing potential treatments for limb-girdle muscular dystrophy type 2A/calpainopathy (LGMD2A), today announced updates to its Gene Therapy Initiative….
Read MoreC3 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 Nationwide Children’s Hospital, to assess the safety and efficacy of adeno-associated viral (AAV)-mediated gene therapy in a mouse model…
Read MoreCoalition 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 Neurology and Neuroscience at the Johns Hopkins School of Medicine, will investigate “Targeting Mss51 as…
Read MoreC3 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 viral (AAV)-mediated gene therapy. This project was funded as a part of the C3 Gene Therapy…
Read MoreDID 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 can have the disease. This form of calpainopathy is called Limb Girdle Muscular Dystrophy type 2A (LGMD2A). In this recessive form, for LGMD2A to develop, two copies of the…
Read MoreDo 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.” As science advanced, it was discovered that many different genetic mutations underlie these conditions. Currently,…
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