Author: Jennifer Levy

Individuals with LGMD are sought for clinical trial evaluating the safety and efficacy of oral weekly glucocorticoid steroids

A clinical trial is being initiated at Northwestern University in Chicago, Illinois. The aim of the study is to test the safety and efficacy of once weekly dosing of oral prednisone, a glucocorticoid steroid. Both ambulatory and nonambulatory patients

Dr. Stefanie Müthel Awarded First Early Career Investigator Travel Grant

Coalition to Cure Calpain 3 (C3) is pleased to announce that Dr. Stefanie Müthel was awarded a Travel Grant for the presentation of her research at the Biology of Calpains in Health and Disease Conference in Pacific Grove, California, from

Patients Sought for C3-funded Clinical Endpoints Study

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