#CureLGMD2A

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

C3 awards grant to University of Florida researchers to investigate calcium handling in LGMD2A/R1

Coalition to Cure Calpain 3 (C3) is driving LGMD2A/R1 research by awarding a new grant to Dr. Elisabeth Barton and Dr. Lan Wei-LaPierre of the University of Florida. The project is titled “Strategies to improve calcium handling in LGMD2A/R1.” LGMD2A/R1

LGMD diagnostic codes proposed for final implementation

BIG NEWS! New diagnostic codes for those living with LGMD that match their diagnosis are likely on their way! Coalition to Cure Calpain 3 has been working with Muscular Dystrophy Association and other LGMD foundations, patients, and expert physicians to

Join Coalition to Cure Calpain 3 in celebrating Rare Disease Day!

Rare Disease Day takes place on the last day of February every year, with the goal of raising awareness about rare diseases and their impact on patients’ lives. Over 300 million people are living with one or more of over

Have you participated in a clinical research study?

Attention Individuals with LGMD2A/R1: Coalition to Cure Calpain 3 wants to hear from you! If you have participated in clinical research, such as a natural history study, an observational study, or a clinical trial, please follow this link to answer

C3 to Participate in Externally-Led PFDD Meeting with FDA

If you are living with LGMD2A/R1, you will have the opportunity to participate in an externally-led Patient-Focused Drug Development Meeting (EL-PFDD) with the FDA on September 23, 2022. C3 is proud to be one of six LGMD organizations to collaborate

Carol Abraham appointed to Patient Engagement Collaborative

Today, the U.S. Food and Drug Administration (FDA), in collaboration with the Clinical Trials Transformation Initiative (CTTI), announced that Carol Abraham has been appointed to the Patient Engagement Collaborative (PEC) and will serve a two year term. Carol is the

Sarepta announces gene therapy program for LGMD2A/R1

Today, Sarepta Therapeutics, Inc. announced that it executed an exclusive license agreement for an LGMD2A/R1 gene therapy program developed by Dr. Zarife Sahenk at Nationwide Children’s Hospital. Preclinical research conducted by Dr. Sahenk provided early proof of concept data for