Research Translating Science into Hope: The CHeT Model for Neurological Drug Discovery By Mark Michaud Nov. 19, 2025 The University of Rochester Center for Health + Technology (CHeT) stands as one of academic medicine’s most successful experiments in translating science into patient benefit. Over nearly four ...
Research Study Shows Hormone Combination Improved Strength and Function in FSHD Patients By Mark Michaud Sep. 8, 2025 A clinical trial found that six months of daily growth hormone plus biweekly testosterone was safe, well-tolerated, and produced meaningful gains in men with facioscapulohumeral muscular dystrophy.
Research Newborn Screening Expansion Sparks Research on Early Duchenne Intervention By Mark Michaud Apr. 21, 2025 A new study will investigate early stage Duchenne muscular dystrophy in infants and toddlers, with the help of newly implemented newborn screening for the disease. The research will enhance ...
Research New Discovery Enables Gene Therapy for Muscular Dystrophies, Other Disorders By Emily Boynton Nov. 14, 2024 Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is a big problem: size. The genes that are dysfunctional in muscular ...
Research Celebrating Gilbert Gottfried: Comedy Stars Unite in NYC to Support Muscular Dystrophy Research By Mark Michaud Nov. 14, 2024 Some of comedy’s biggest names gathered in New York City on Wednesday to honor the life of Gilbert Gottfried and raise funds for muscular dystrophy research at the University of Rochester Medical ...
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: URMC neuromuscular 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.