C3 News

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

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

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

C3 partners with 16 LGMD organizations in letter to FDA

Coalition to Cure Calpain 3 is proud to be one of 17 organizations dedicated to LGMD to have collaborated on a statement submitted to the Food and Drug Administration in conjunction with the recent Patient-Focused Drug Development Meeting Patient Perspectives

Our GIVING TUESDAY fundraiser starts NOW!

Champion Coalition to Cure Calpain 3’s cause to raise $50,000 for LGMD2A/R1 gene therapy research. Every dollar donated between now and December 1, 2022 will be matched, up to $25,000 by a family motivated to drive a gene therapy cure.

New LGMD2A/R1 Mice: Novel tools for drug development

Coalition to Cure Calpain 3 (C3) is excited to announce the generation of four mouse models of limb-girdle muscular dystrophy type 2A (LGMD2A/R1). These mouse models were developed and characterized as part of a research grant from C3 to Dr.