Journal of Genetic Syndromes & Gene Therapy
Open Access

Abstract

Report: Fourth International Workshop for Glycosylation Defects in Muscular Dystrophies

Anthony Blaeser, Amy Harper, Kevin Campbell and Qi Long Lu

The Fourth International Workshop for Glycosylation Defects in Muscular Dystrophies took place on April 16- 17, 2015 at the Fairfield Inn and Suites Charlotte Uptown, Charlotte, North Carolina. The workshop was hosted by the McColl-Lockwood Laboratory for Muscular Dystrophy Research, and sponsored by the Carolinas HealthCare Foundation, the Muscular Dystrophy Association (MDA), funds raised by “Riding 4 Research” and generous support from the McColl and Lockwood families. Clinicians and scientists from the US, UK, Germany and Japan presented a total of 21 talks spread out over 2 days. The workshop was divided into three sessions: Session A focused on the current status of the development of animal models for diseases caused by defects in muscle-protein glycosylation, on our current understanding of such glycosylation and on the mechanisms that lead to disease. Session B focused on preclinical therapeutics, in particular on AAV- and drug-based therapies. Session C covered the clinical management of muscular dystrophies and endpoint evaluation.