Author: Jennifer Levy

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

Myonexus Therapeutics announces initiation of a gene therapy clinical trial for LGMD2E

On November 8, 2018, Myonexus Therapeutics announced the initiation of a clinical trial evaluating MYO-101, a novel gene therapy candidate for beta-sarcoglycanopathy, also known as limb girdle muscular dystrophy type 2E (LGMD2E). The trial is underway at Nationwide Children’s Hospital’s

C3 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

C3 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