Coalition to Cure Calpain 3 (C3) attended the 23rd International Annual Congress of the World Muscle Society, October 2-6 2018 in Mendoza, Argentina. Dr. Jennifer Levy, C3 Scientific Director, represented C3 and presented a poster detailing our mission, registry, and research grants program. Attendees include neuromuscular clinicians, academics, research scientists, and industry leaders from around the world. The Congress covered a variety of topics related to advances across the neuromuscular field, including development of treatments of neuromuscular disorders. Among the highlights:
- R. El-Khoury, American University of Beirut Medical Center, Lebanon, showed data on the different mitochondria deficiencies observed in muscle biopsy samples from LGMD2A patients
- Conrad Weihl, Washington University St. Louis, USA, demonstrated progress towards identifying the symptoms that affect the health and daily function of individuals with LGMD using a qualitative, patient-directed approach
- Jerry Mendell presented updated results from Sarepta Therapeutics gene therapy micro-dystrophin trial to treat patients with Duchenne Muscular Dystrophy. They have observed efficient gene transfer and a favorable safety profile. Early results suggest functional improvements for the patients, however, this is a small data set and results still need to be confirmed with a larger trial
The next World Muscle Society Congress will be held October 1-5, 2019 in Copenhagen, Denmark.