Coalition to Cure Calpain 3 (C3) is pleased to share that Dr. Volker Straub has been appointed to our Scientific Advisory Board (SAB), joining Dr. Melissa Spencer (Chair), Dr. Kevin Campbell, Dr. Eric Hoffman, and Dr. Louis Kunkel.
Read MoreKNOW YOUR CODE: Why the new ICD-10 codes matter, and what you can do to confirm your records
Did you know that new ICD-10 codes were assigned for limb-girdle muscular dystrophies (LGMDs) in October 2022? Patients with LGMD2A/R1, autosomal recessive LGMD due to Calpain 3-dysfunction, have been given a diagnosis code of G71.032. Patients with LGMD1i/D4, autosomal dominant LGMD due to Calpain 3-dysfunction, are grouped with other autosomal dominant LGMDs with a diagnosis…
Read MoreGenetic 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, it was discovered that many different genetic variants underlie these conditions. Currently, there are more…
Read MoreDr. 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 3-7, 2023. The goal of the C3 Travel Grant program is to foster early career…
Read MoreCoalition to Cure Calpain 3 in partnership with the National Organization forRare 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 form of muscular dystrophy that causes progressive muscle weakness and wasting. Calpainopathy currently has no…
Read MoreNew 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).
Read MoreC3 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 Walton Muscular Dystrophy Research Centre (JWMDRC) at Newcastle University. The project, titled “Improving clinical trial readiness by investigating the suitability of clinical outcome assessments and documenting the…
Read MorePublication 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 model of LGMD2A/R1. Zebrafish are often used to study genetic disorders because they grow and…
Read MoreNew 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 project,“A diagnostic functional test to rule out dominant forms of calpainopathy,” will develop a tool to…
Read MoreC3 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 on Gene Therapy Products. Special thanks to The Speak Foundation, Joshua Thayer Esq., and Bradley Williams…
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