Coalition to Cure Calpain 3 (C3) is pleased to announce that a research grant has been awarded to Dr. Nicholas Johnson, Associate Professor & Vice Chair of Research, Virginia Commonwealth University. The project is titled “Defining Clinical Endpoints in LGMD.”
C3 Hosts Gene Therapy for LGMD2A Workshop
Coalition to Cure Calpain 3 (C3) is committed to treating and ultimately curing limb-girdle muscular dystrophy, type 2A (LGMD2A), a form of calpainopathy. C3’s mission is to fund therapeutically relevant research and clinical trials as we educate the global community about
May 20 is Clinical Trials Awareness Day
It is CLINICAL TRIALS AWARENESS day… C3 often gets asks, are there any clinical trials underway for LGMD2A (a form of calpainopathy)? Yes! One form of clinical trial, called a natural history study, is currently underway. This type of study
Sarepta Therapeutics adds LGMD2A to their pipeline
Today, Sarepta Therapeutics, Inc. announced that it has signed an agreement with Nationwide Children’s Hospital giving Sarepta the exclusive option to Nationwide’s gene therapy candidate to treat limb girdle muscular dystrophy type 2A (LGMD2A), a form of calpainopathy. This program
C3 Awards Research Grant to Dr. Jaakko Sarparanta to Investigate Relationship Between Calpain 3 Activity and the Protein Titin
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
Sarepta Therapeutics Announces Preliminary Results in Clinical Trial Investigating Gene Therapy for LGMD2E
Today Sarepta Therapeutics announced preliminary data on the first three LGMD2E patients dosed in the MYO-101 Gene Therapy Trial. Muscle biopsies show that about 50% of muscle fibers show expression of beta-sarcoglycan, the protein which is missing or faulty in
New 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
C3’s 2018 Year in Review
Happy New Year! We at Coalition to Cure Calpain 3 (C3) thank our Board of Directors for working alongside us and our generous donors for helping us realize these achievements in 2018: THREE NEW RESEARCH GRANTS AWARDED IN 2018 Development of Gene
SAVE THE DATE: National LGMD Conference to be held August 30 – September 2, 2019
The National Limb Girdle Muscular Dystrophy Conference will be held in Chicago, Illinois on August 30 – September 2, 2019 at the Hyatt Regency. The conference is dedicated to bringing every patient into an active role seeking to find cures
LGMD2A Patients Sought for Natural History Study at Nationwide Children’s Hospital
A natural history study of limb girdle muscular dystrophy type 2A (LGMD2A, a form of calpainopathy) has been initiated by Linda Lowes, PT, PhD at Nationwide Children’s Hospital in Columbus, Ohio. The aim of this study is to observe
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