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.
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 URMC Communications 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, ...
