The KU neuromuscular division is directed by Dr. Mazen M. Dimachkie, an international leader in neuromuscular disease research. Dr. Dimachkie is associate director for the KUMC NeuroNext site, named Heartland Unit for Neuroscience Trials (HUNT) and the Vice-Chairman for Research Programs at the Neurology Department of the KUMC. He is also associate director of the Institute for Neurologic Discoveries. Since joining the KUMC in 2007, he has been directly involved in all aspects, activities and processes relating to the KU NMRD and the arimoclomol in inclusion body myositis (IBM) pilot study. He is the PI pn the the currently funded 1RO1FD004809-01A2 multicenter international randomized controlled trial investigating the efficacy and safety of Arimoclomol in IBM. He is the overall sponsor and study PI for a multicenter North American study of SCIg in myasthenia gravis funded by CSL-Behring. He is also involved in a variety of federally-funded neuromuscular research projects as well pharma-sponsored studies, nationally and internationally. I currently serve since 2013 as. He has research experience in a broad range of neuromuscular disorders including ALS, PLS, SMA, myasthenia gravis, the Lambert-Eaton myasthenic syndrome, Pompe disease, muscular dystrophies, myositis, neuropathies and ALS. The KU NMRD is an active site in national consortia (Western ALS consortium, Northeast ALS consortium, ALS Research Group, and Muscle Study Group).
Dr. Barohn is an international leader in neuromuscular disease research, who is chairman of the Neurology Department and Vice Chancellor for Research at the KUMC. Dr. Barohn has active pharmaceutical, foundation, and NIH-sponsored studies in a variety of neuromuscular diseases. Dr. Barohn was the Co-PI of the Consortium for Clinical Investigations of Neurological Channelopathies (CINCH), which is part of the NIH Rare Disease Clinical Research Network (RDCRN). Dr. Barohn also has valuable experience in leading ongoing multi-site clinical studies. A Mexilitene in Non-Dystrophic Myotonia Study was funded in 2008 (R01 FD003454, PI Barohn) and had 7 sites in the US, England, and Italy. A Methotrexate in Myasthenia Gravis study was funded in 2009 (R01FD003538, PI Barohn) and has 16 sites in the US and Canada and the FDA-OPD funded North American study of Rasagiline in ALS as well as the PCORI-funded study PAIN-CONTROLS.
The KU NMRD team additionally includes five faculty members, 7 research coordinators, one clinical evaluator, and three project manager, and manages approximately 64 active trials at any given time with half of these investigator-initiated and half industry-sponsored. In addition to Drs. Dimachkie and Barohn, the KU NMRD consists of other faculty members in the department of neurology that form a team that is engaged in neuromuscular clinical trials and active neuromuscular disease clinics. Other investigators on the team include Mamatha Pasnoor, MD (associate professor of neurology and the co-director of the KU neuropathy center), Melanie Glenn, MD (clinical assistant professor), Omar Jawdat, MD (assistant professor and ALSA clinic director), Jeffrey Statland, MD (assistant professor and MDA clinic co-director), and Laura Herbelin, BSc (clinical instructor and project manager).