#CureCalpain3

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.

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.

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

Limb-girdle muscular dystrophy coalition to host externally-led patient focused drug development meeting

C3 is excited to announce that we’ve teamed up with the LGMD2D Foundation, the Kurt+Peter Foundation, CureLGMD2I, the McColl-Lockwood Laboratory for Muscular Dystrophy Research, and the Speak Foundation to host an Externally-Led Patient Focused Drug Development (EL-PFDD) meeting with the

C3 funds development of new LGMD2A/R1 disease model for preclinical testing

Coalition to Cure Calpain 3 (C3) is pleased to announce the funding of a new research award to Dr. Pia Elustondo of AGADA Biosciences. This project will characterize a novel LGMD2A/R1 disease model and utilize this model to test several