Uncategorized

Trial readiness study for individuals with LGMD2A/R1

Please contact either Ruby Langeslay or Jennifer Raymond at Virginia Commonwealth University for more information

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

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

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

Travel Grant Program for Early Career Investigators

Coalition to Cure Calpain 3 will award Travel Grants to foster early career investigators who are currently conducting research in the areas of limb-girdle muscular dystrophy type 2A (LGMD2A/R1) or Calpain 3. The grants support travel expenses to attend relevant

Together we are STRONGER: 8th Annual LGMD Awareness Day is coming up!

There is great strength in awareness, understanding, and unity. The LGMD community demonstrates global connectivity each year when we campaign together and gain recognition for LGMD. With over 30 different genetic types of LGMD, the act of collaborating globally makes

Pre-registration is now open for the LGMD EL-PFDD Meeting with FDA and other stakeholders

PRE-REGISTER NOW FOR THE LGMD EXTERNALLY-LED PATIENT-FOCUSED DRUG DEVELOPMENT MEETING TO BE HELD ON FRIDAY, SEPTEMBER 23. This no-cost, public virtual meeting is open to those living with LGMD2A, their families and caregivers, physicians, clinicians, industry/pharma representatives, academic researchers, FDA

Published Research Alert: CAPN3 gene therapy improves muscles function in a mouse model of limb-girdle muscular dystrophy type 2A/R1 (LGMD2A/R1)

Coalition to Cure Calpain 3 (C3) is pleased to announce the publication of important research undertaken by Dr. Zarife Sahenk, Dr. Jerry Mendell, and colleagues at Nationwide Children’s Hospital in Columbus, Ohio. The paper, titled “Systemic delivery of AAVrh74.tMCK.hCAPN3 rescues the

LGMD2A/R1 Participants Sought for COVID-19 Survey

Coalition to Cure Calpain 3 is sharing the following communication from the University of Rochester regarding a voluntary research survey to be completed by those living with LGMD2A/R1, a form of calpainopathy, or by their caregivers. Study Investigator: Rabi Tawil, MD

« Previous