title: On the path to a Duchenne muscular dystrophy therapy.
abstract: Duchenne Muscular Dystrophy (DMD) is a devastating inherited disease of children with no effective therapies. Here I discuss the landscape for new treatments and the history, current status and prospects for our work developing recombinant biglycan as DMD therapy.
authors: Professor of neuroscience at Brown University and cofounder of Tivorsan Pharmaceuticals.
authors: Justin
authors: J
authors: Fallon
mesh_terms: D000690:Amyotrophic Lateral Sclerosis / Q000188:drug therapy* / Q000235:genetics / Q000503:physiopathology; D058578:Biglycan / Q000494:pharmacology*; D035843:Biomedical Research / Q000639:trends; D003598:Cytoskeletal Proteins / Q000187:drug effects* / Q000378:metabolism; D015195:Drug Design; D004345:Drug Industry / Q000639:trends; D016189:Dystrophin / Q000187:drug effects* / Q000378:metabolism; D005091:Exons / Q000187:drug effects; D006801:Humans; D058990:Molecular Targeted Therapy / Q000639:trends; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy* / Q000235:genetics / Q000503:physiopathology; D011401:Promoter Regions, Genetic; D012237:Rhode Island; D049411:Utrophin / Q000187:drug effects* / Q000378:metabolism
chemical_list: D058578:Biglycan; D003598:Cytoskeletal Proteins; D016189:Dystrophin; D049411:Utrophin
keywords: Biglycan; Duchenne Muscular Dystrophy; neuromuscular disorders