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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

FDA Patient Listening Session on LGMDs held October 20, 2020

On October 20th, 2020, the LGMD community was able to meet with the FDA to share our patient experiences. This was a patient-led listening session organized by a consortium of advocacy organizations, including Coalition to Cure Calpain 3. The session

C3 is Putting LGMD on the Map!

Coalition to Cure Calpain 3 is putting limb-girdle muscular dystrophy type 2A (LGMD2A/R1) on the map! In celebration of ten years of progress in our mission to fund research for a cure and raise global awareness, C3 is proud to

Dr. Timothy Dalton Spins for a Cure!

We are grateful to Dr. Timothy Dalton for generously donating his winnings of $7,700 from his March 2, 2020 appearance on the popular television game show “Wheel of Fortune” to C3! As a non-profit organization dedicated to funding research efforts

LGMD2A Patients Sought for Natural History Study at Nationwide Children’s Hospital

A natural history study of limb girdle muscular dystrophy type 2A (LGMD2A, a form of calpainopathy) has been initiated by Linda Lowes, PT, PhD at Nationwide Children’s Hospital in Columbus, Ohio. The aim of this study is to observe

Myonexus Therapeutics announces initiation of a gene therapy clinical trial for LGMD2E

On November 8, 2018, Myonexus Therapeutics announced the initiation of a clinical trial evaluating MYO-101, a novel gene therapy candidate for beta-sarcoglycanopathy, also known as limb girdle muscular dystrophy type 2E (LGMD2E). The trial is underway at Nationwide Children’s Hospital’s

FDA to step up enforcement of stem cell clinics

Stem cells are cells that have the unique ability to self-renew and to transform into many specialized cell types. They can be isolated either from embryos or from adult tissue. Adult stem cells are being used for various medical therapies

Rare disease stakeholders connect at 2017 RARE Patient Advocacy Symposium

Rare disease patients, caregivers, representatives from advocacy organizations, and other rare disease stakeholders met on May 19, 2017 in Philadelphia for the 2017 RARE Patient Advocacy Symposium, a partnership of Global Genes and the Penn Medicine Orphan Disease Center. The