Author: Jennifer Levy

Countdown to the New Year – 2023 Edition

This year, C3 collaborated with 5 other LGMD organizations to publish the Voice of the Patient Report, summarizing the 2022 Externally-Led Patient Focused Drug Development Meeting and highlighting the unmet medical needs of the LGMD community. READ MORE Connected with

New LGMD2A/R1 mouse model available at Jackson Labs

Coalition to Cure Calpain 3 (C3) is pleased to share that a new Calpain 3-null mouse strain is available through the Jackson Labs repository. This line is crossed onto an NSG background, resulting in an immunodeficient strain. The NSG.Capn3 KO mice may

Genetic testing for LGMDs

Limb-girdle muscular dystrophies (LGMDs) were characterized in the clinic before anyone knew what caused the diseases. Consequently, several different diseases that all looked the same to physicians were lumped together into one category that they called “LGMD.” As science advanced,

Dr. Stephanie Hunn awarded C3 Travel Grant

Coalition to Cure Calpain 3 (C3) is pleased to announce that Stephanie Hunn DPT was awarded a Travel Grant for the presentation of her research at the Annual Congress of the World Muscle Society in Charleston, South Carolina, from October

Coalition to Cure Calpain 3 in partnership with the National Organization for
Rare Disorders (NORD®) launches LGMD2A/Calpainopathy Registry

Today, Coalition to Cure Calpain 3 (C3) in partnership with the National Organization for Rare Disorders (NORD®) launched a study with global reach to research Calpainopathy (including limb-girdle muscular dystrophy type 2A/LGMDR1 and limb-girdle muscular dystrophy type 1I/LGMDD4), a rare

Vita Therapeutics enters licensing agreement with MilliporeSigma

Today, Vita Therapeutics announced they entered a licensing agreement with MilliporeSigma to advance their cell therapy program to treat LGMD2A/R1. This agreement allows Vita to utilize MilliporeSigma’s CRISPR gene editing patents to insert a functional copy of the CAPN3 gene

May 20 is Clinical Trials Day

On this day, C3 would like to take the opportunity to discuss the DRUG DEVELOPMENT PROCESS for rare diseases such as LGMD2A/R1. DISCOVERY: Drug development usually begins in the laboratory, where researchers seek to gain new insights into a disease process. With a

New “Voice of the Patient” report highlights unmet medical needs of six subtypes of LGMD, including LGMD2A/R1

Today, Coalition to Cure Calpain 3 joins five other LGMD patient organizations (collectively referred to as The LGMD Coalition*) to release the ‘Voice of the Patient Report’ summarizing outcomes from the September 2022 Externally-Led Patient Focused Drug Development Meeting (EL-PFDD).

C3 funds retrospective analysis of clinical outcome assessments

Coalition to Cure Calpain 3 (C3) is pleased to announce the funding of a new research award to Dr. Meredith James and Professor Jordi Díaz-Manera of the John Walter Muscular Dystrophy Research Centre (JWMDRC) at Newcastle University. The project, titled

Publication shares development of new animal model of LGMD2A/R1

A paper published by Jason Berman, Children’s Hospital of Eastern Ontario Research Institute and University of Ottawa, along with colleagues from Dalhousie University, AGADA Biosciences, and Binghamton University – State University of New York, shares the development of a zebrafish