Jane Buckner, MD and Carla Greenbaum, MD | Mar 1, 2023 | 4 min read
A therapy for type 1 diabetes is the first to treat patients before symptoms appear, paving the way toward preventing this and other autoimmune diseases.
A panel of experts advised the US Food and Drug Administration that BioMarin Pharmaceutical has not demonstrated efficacy of its new drug for Duchenne muscular dystrophy.