Unwilling to accept the slow pace of progress in CMT drug development, the CMT Research Foundation has launched a new video series that asks and answers the most pressing questions about CMT treatments and drug development.
In this first episode hosted by CMT Research Foundation CEO Susan Ruediger, leading neurologists and geneticists will have you on the edge of your seat as they share when patients can expect treatments for CMT and how we can be the “special sauce” in accelerating progress.
After watching this video, you will have answers to these questions:
- Why is it taking so long to deliver treatments for CMT, especially when other diseases like multiple sclerosis and spinal muscular atrophy are bringing so many drugs to market?
- Why are patients and patient-led foundations important in drug development, and what can we do now to speed up the process?
- When can people with CMT expect treatments?
Meet the Experts
Get to know the CMT research experts featured in this video:
Dr. Chris Austin, Director, National Center for Advancing Translational Sciences, National Institutes of Health
Dr. Austin has served as director of the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health since 2012. Prior to this role, he was NCATS’ scientific director, focusing on translating basic science discoveries into new treatments and technologies to improve the efficiency of therapeutic/diagnostic development. He founded several initiatives, including the NIH Chemical Genomics Center, the Therapeutics for Rare and Neglected Diseases program and the Toxicology in the 21st Century program. Before joining NIH in 2002, he led genomic-based target discovery, pharmacogenomi, and neuropsychiatric drug-development programs at Merck. From 2016 to 2018, he served as chair of the International Rare Disease Research Consortium (IRDiRC). Dr. Austin is also a member of National Academy of Medicine. He earned an A.B. from Princeton University, an M.D. from Harvard Medical School and completed training in internal medicine and neurology at Massachusetts General Hospital.
Dr. Sindhu Ramchandren, Medical Director of Neurology, PRA Health Sciences
Dr. Sindhu Ramchandren is a board-certified neurologist who joined PRA Health Sciences in March 2018 — a global clinical research organization. Her role is to provide innovative solutions to move drug discovery forward and bring more therapies to the market for patients with rare neuromuscular disorders. Before joining PRA, Dr. Ramchandren was the medical director of the Muscular Dystrophy Association Care Center and the CMT Center of Excellence at the University of Michigan and an integral member of the Inherited Neuropathy Consortium. Her clinical research focused on patient-centered outcomes, including quality of life measures, for use in clinical trials in CMT and muscular dystrophies. Dr. Ramchandren completed her medical degree at the University of Texas-Houston, neurology residency at the University of Pennsylvania, clinical neurophysiology-EMG fellowship at Johns Hopkins University, and her master’s of science in clinical research design and statistical analysis at the University of Michigan.
Thank you to our partners at AcuraStem and Shift Pharmaceuticals for sponsoring this video and underwriting the costs to make it possible.