People Tawil’s Retirement Marks Milestone in Neuromuscular Disease Research and Treatment By Mark Michaud Jun. 17, 2024 Rabi Tawil, MD, one of the world’s leading experts in rare neuromuscular diseases, is retiring after more than three decades with URMC.
Research RNA Center of Excellence to Develop New Treatments, Train Future Scientists By Emily Boynton May. 9, 2024 The field of RNA biology has catapulted to the forefront of health and medicine over the past decade as the work of scientists like Lynne E. Maquat, PhD, director of the University of Rochester ...
Research Calcium Channel Blockers Key to Reversing Myotonic Dystrophy Muscle Weakness, Study Finds By Mark Michaud Jan. 2, 2024 The study demonstrates that calcium and chloride channelopathy lies at the heart of the muscle dysfunction found in the disease and that common clinically available calcium channel blockers can ...
Research From lab to life-changer: 鶹Ƶneuromuscular researchers pave the way for gene therapy revolution By Mark Michaud Oct. 23, 2023 For almost five decades, researchers with the Medical Center have been studying myotonic dystrophy, Duchenne muscular dystrophy, and other rare neuromuscular disorders, and played a pivotal role in ...
Research Grant Boosts Drive to Transform Treatment Landscape for Rare Neurological Disorders By Mark Michaud Aug. 28, 2023 New funding will extend URMC's key role in national research network just a new gene therapies or neuromuscular and other disorders are emerging.
Patient Care New Gene Therapy for Duchenne Muscular Dystrophy a “Monumental Advance” By Mark Michaud Jun. 26, 2023 Emma Ciafaloni, MD, describes how the new drug works, why the FDA chose to limit its use, and what it means for patients and their families.
Not forgotten: hospital physicians work to treat, study the rarest of diseases By 鶹ƵCommunications Jul. 27, 2016 Since April 2015, Nicholas O’Neill has traveled nearly three hours by car every week from southeast of Syracuse to Golisano Children’s Hospital. Here, in the confines of the Clinical Research Center, ...