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, ...
