C3 News

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.

Your voice made a difference!

THANK YOU to all those families impacted by limb-girdle muscular dystrophy types 2A, 2C, 2D, 2E, 2F, and 2I who made their voices heard at the LGMD EL-PFDD! Whether you were on camera, called in, submitted written comments, or participated

The LGMD EL-PFDD is just one week away!

Join us for this important LGMD event on September 23rd beginning at 10:00 AM EDT and make YOUR VOICE HEARD. Bookmark www.lgmdpfdd.com so you can view and participate live.

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

Casimir Recruiting LGMD2A/R1 Patients for At-Home Study

The Casimir LGVA study is quickly filling! We are specifically seeking individuals with LGMD2A who can walk across a room and stand up from sitting on a couch to participate. Email LGMDS@casimirtrials.com or call (800)542-0948 for more information.