This translational research program is focused on bridging the gap between clinical care and the basic sciences in neuromuscular disorders.
The program was officially formed in October 2012 under the leadership of Dr. Richard Nowak. While the program is based within the Department of Neurology and rooted in the Division of Neuromuscular Medicine, we are committed to collaboration across many disciplines, such as immunology, genetics and biochemistry. Our primary research focus has been on immune-mediated neuromuscular diseases, such as myasthenia gravis (MG) and inflammatory neuropathies and myopathies (i.e., Guillain-Barré syndrome (GBS), chronic inflammatory demyelinating polyneuropathy (CIDP), Inclusion Body Myositis). We believe that fostering close collaboration between clinicians and basic scientists is critical in successfully advancing our understanding of these disorders and translating these discoveries into clinical practice. Biomarker discovery and working to the end of patient-tailored treatment strategy development is our major overall objective.
Our program is also a part of the Muscle Study Group (MSG), which is a consortium of scientific investigators among academic and research centers committed to the cooperative planning, implementation, analysis and reporting of controlled clinical trials and of other research for muscle and other neuromuscular diseases.
Since formation of this program, we have initiated eight studies, five of which are focused on MG. Early observations made by our group and others showing the benefit of B cell targeted therapy (rituximab) for MG has compelled us to explore this further. The success of this program is highlighted by the fact that Dr. Nowak was awarded a 4.8 million dollar grant in 2013 to conduct the first NIH-sponsored clinical trial in MG and is the national principal investigator for the multi-center study entitled A Phase 2 Trial of Rituximab in Myasthenia Gravis.