#CureLGMDR1

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.

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

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

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

FUNDING OPPORTUNITY! Coalition to Cure Calpain 3 Issues Request for Applications for Research Related to LGMD2A/R1

Coalition to Cure Calpain 3 requests applications for research and translational projects related to calpain 3 and limb-girdle muscular dystrophy type 2A (LGMD2A, also called LGMDR1 Calpain 3-related). The goal of the RFA is to enable the development of therapeutic

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

New publication illuminates the effects of the COVID-19 pandemic on people with muscular dystrophy

A new publication examines the social and health impacts of the COVID-19 pandemic and the associated social guidelines on people living with muscular dystrophies. The authors developed a COVID-19 Impact Survey for adults living with facioscapulohumeral muscular dystrophy, myotonic dystrophy,

Will YOU help advance LGMD2A/R1 research by participating in the GRASP-LGMD Natural History Study?

Natural history studies collect information about the symptoms and progression of a disease in the absence of an intervention. It is vital to understand natural history so that researchers can plan for clinical trials to determine if an investigational drug