|Education||MS, Johns Hopkins University|
A Xenograft Model for Facioscapulohumeral Muscular Dystrophy
Despite being one of the most prevalent neuromuscular disorders worldwide, there have been very few clinical trials in Facioscapulohumeral muscular dystrophy (FSHD). Currently there are no clinical trials and no good therapeutic options for this progressively disabling disease. One of the main impediments to the development of novel drugs for FSHD is the lack of an accepted animal model stemming from an incomplete understanding of the pathogenesis of the disease. We are therefore developing a novel animal model which is independent of any hypothesis of pathogenesis.