What is a natural history study? A natural history study is a research study that collects information about the genetics, symptoms, and lived experiences of individuals. These studies are considered observational, because they do not include an interventional drug or therapy,
C3 is delighted to share that a research grant has been awarded to Dr. Peter Davies of Queen’s University in Ontario, Canada. The project, titled “Solving the structure of Calpain 3 and assessing the impact of LGMD2A/R1,” aims to determine
C3 is excited to share that a research grant has been awarded to Dr. Svetlana Gorokhova and Dr. Marc Bartoli, Translational Neuromyology Team, Marseille Medical Genetics Institute at Aix Marseille University. The project, “Optimizing the functional assay to identify novel
Coalition to Cure Calpain 3 (C3) is driving LGMD2A/R1 research by awarding a new grant to Dr. Elisabeth Barton, Dr. Lan Wei-LaPierre, and Dr. Siobhan Malany of the University of Florida. The project is titled “Store-operated calcium entry modulation as
C3 is pleased to announce the funding of a new research project to Dr. Christopher Heier, of Virginia Commonwealth University in the USA, and Dr. Utkarsh Dang, of Carleton University in Canada. The project is titled “Comparative proteomics of LGMD2A/R1
A new paper, “Motor Function in LGMD2A/R1: Validation of Clinical Outcome Assessments for Clinical Care and Trial Readiness,” was recently published in the journal Neurology: Genetics. This important, C3-funded initiative is a testament to international cooperation, bringing together leading LGMD
The 2025 International LGMD Conference, hosted by the Speak Foundation, was attended by over 500 LGMD patients and family members, clinicians, and scientists. For the first time, the conference included a scientific poster session for researchers to present their basic, translational, or
New data from the GRASP-001 study identify clinical outcome assessments for future LGMD2A/R1 clinical trials A team of LGMD researchers, called the GRASP-LGMD Consortium, published the baseline results of their LGMD2A/R1 clinical outcome study in the Annals of Clinical and Translational Neurology.
Muscular Dystrophy Association (MDA) and Coalition to Cure Calpain 3 (C3) partner to fund $300,000 research grant for gene therapy development in limb-girdle muscular dystrophy. This grant supports the work of Melissa Spencer, PhD, Director of the Neuromuscular Division in
SYNOPSIS Coalition to Cure Calpain 3 (C3) provides funding for research and translational projects to expand the understanding of calpain 3 and Calpainopathy (including LGMD2A/R1 and LGMD1I/D4) to enable development of therapeutic approaches for this disease. This award is intended