Neuromuscular Diseases

Research

Drs. Weiss and Distad are currently site investigators for two ALS clinical treatment trials. These include a phase II multicenter study comparing the effects of high dose creatine and tamoxifen and a phase III multicenter study investigating the effects of dexpramipexole compared to placebo. They are also involved in a multicenter study investing the response to intravenous immunoglobuliun therapy in the treatment of multifocal motor neuropathy with conduction block. Dr. Distad is also the site coordinator for the multicenter Thymectomy in Myasthenia Gravis study, comparing the treatment of thymectomy with medication alone.

A number of affiliated basic and clinical researchers at the University of Washington have a strong interest in neuromuscular diseases. Stephen Tapscott, MD, PhD, at the Fred Hutchinson Research Center, has a particular focus in understanding the pathogenic mechanisms of myotonic dystrophy type 2 and facioscapulohumeral muscular dystrophy (FSHD) as well as potential therapies for these conditions. Jeffrey Chamberlain, PhD, is an internationally renowned neuroscientist with an interest in the pathophysiology of Duchenne muscular dystrophy and the use of gene therapies in slowing the condition, employing a mouse disease model. Thomas Bird, MD, is a renowned neurogeneticist whose research focuses primarily on genetic studies of Charcot Marie Tooth hereditary neuropathy and clinical studies of FSH muscular dystrophy. Joel Chamberlain, PhD, like her husband Jeffrey, has a strong interest in understanding and treating muscular dystrophies. Her research is concentrated on the use of RNA interference in the treatment of myotonic dystrophy type 1, employing a mouse model for the disease.