Coalition to Cure Calpain 3

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

Join Coalition to Cure Calpain 3 in celebrating Rare Disease Day

Rare Disease Day takes place on the last day of February every year, with the goal of raising awareness about rare diseases and their impact on patients’ lives. Over 300 million people are living with one or more of over

Sarepta Community Update on LGMD programs

Sarepta Therapeutics recently published a Community Letter to share updates on their limb-girdle muscular dystrophy gene therapy programs. Click here to access the letter on Sarepta’s website.

New research grant aims to improve genetic diagnoses in individuals with CAPN3 mutations

Coalition to Cure Calpain 3 (C3) is pleased to announce a research grant has been awarded to Dr. Svetlana Gorokhova, working together with Dr. Marc Bartoli in the Translational Neuromyology Team, Marseille Medical Genetics Institute at Aix Marseille University. The

C3’s Countdown to the New Year

In 2022, C3 … Announced 5 new research projects Generated 4 novel mouse models for preclinical drug testing Co-organized an EL-PFDD meeting where 300+ individuals with LGMD tuned in to share their perspectives on what it is like to live