Community Webinar: NS Pharma Exon Skipping Trials Brogidirsen and NS-050/NCNP-03

Dr. Clemens is Professor and Vice Chair of Neurology at the University of Pittsburgh and Chief of the Neurology Service at Pittsburgh VA Healthcare System.

Dr. Clemens is an adult neurologist who has scientific expertise in translational and clinical research developing therapies for skeletal muscle disorders caused by genetic mutations such as Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD) and late-onset Pompe disease. Dr. Clemens contributed to the research sponsored by NS Pharma that led to regulatory approval of a morpholino-based exon 53 skipping therapy for the treatment of patients with DMD whose mutation is amenable to skipping exon 53 of the dystrophin gene.

Dr. Clemens led the Cooperative International Neuromuscular Research Group (CINRG) as Medical Director from 2010-2023. She has chaired several multi-site studies conducted by the CINRG network, including an international, longitudinal natural history study of BMD and a series of treatment studies that led to regulatory approval of a novel steroid for the treatment of DMD. Dr. Clemens led research at one of 5 sites worldwide resulting in regulatory approval of the first recombinant enzyme treatment for late-onset Pompe disease. She contributed to the research leading to the next generation of recombinant enzyme therapy for this disorder. She serves on the North American Board for the Pompe Registry.