After a two year campaign, those with LGMD will now have a diagnostic code that matches their diagnosis! C3 is proud to have played a role in this victory alongside our friends and advocacy partners, including the Muscular Dystrophy Association, CureLGMD2i, Jain Foundation, Kurt+Peter Foundation, LGMD Awareness Foundation, LGMD1D DNAJB6 Foundation, LGMD2i Research Fund, and…
Read MoreC3 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 candidate drugs. Preclinical drug screening is one of the critical initial steps to develop new…
Read MoreC3 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 is caused by mutations in the gene for Calpain 3. Researchers do not fully understand…
Read MoreWhat’s on the Horizon for LGMD2A/R1?
Dr. Jennifer Levy, Coalition to Cure Calpain 3 Scientific Director, is a proud contributor to the Winter 2022 issue of LGMD News. This magazine, a project of The Speak Foundation, publishes up-to-date information for the LGMD community. Dr. Levy’s article shares updates on the different therapeutic approaches being pursued to develop a treatment for LGMD2A/R1. ACCESS THE…
Read MorePublished 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 phenotype in a mouse model for LGMD2A/R1,” was published in Molecular Therapy: Methods &…
Read MoreC3 Awards Research Grant to Drs. Melissa Spencer and Jeffrey Chamberlain to Develop Gene Therapy Vectors for LGMD2A/R1
Coalition to Cure Calpain 3 (C3) is delighted to announce a newly-funded research grant to develop a gene therapy for LGMD2A/R1 using vectors optimized for gene delivery to muscles most affected by the disease. This work involves a multi-lab collaboration between Dr. Melissa Spencer at the University of California Los Angeles and Drs. Jeffrey Chamberlain…
Read MoreC3 Selected as Platinum Member of the National Organization for Rare Diseases
Coalition to Cure Calpain 3 (C3) has joined the National Organization for Rare Diseases (NORD) as a Platinum Member. This level of membership represents the highest level of integrity and transparency for patient advocacy organizations involved in medical research, drug development, medical education, and registries. NORD is an independent advocacy organization representing the approximately 25-30 million Americans…
Read MoreFDA 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 focused on various aspects and genetic subtypes of LGMD. Fifteen presenters, all patients or family…
Read MorePromising study identifies compound that improves muscle in LGMD2A/R1 mice
A new publication from Dr. Melissa Spencer’s laboratory at the University of California Los Angeles identifies a compound that mimics the effects of exercise in a mouse model of limb girdle muscular dystrophy type 2A (LGMD2A/R1). These studies may point the way to similar compounds that will benefit people living with muscular dystrophy. Mutations…
Read MoreNotes from 23rd annual meeting of the American Society for Gene and Cell Therapy
The American Society for Gene and Cell Therapy (ASGCT) annual meeting kicked off on Tuesday, May 12, 2020. Due to COVID-19, the meeting was held virtually. This meeting covers the field’s latest and most innovative science across all diseases and conditions. Several sessions were dedicated to muscular dystrophies, and four presentations focused on animal and…
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