by Madaline Spencer | Nov 14, 2024
Judith Thompson, Rare Disease Population Health Strategy Lead at UCB, discusses social determinants of health in rare diseases. As Ms. Thompson explains, social determinants of health are the conditions in which people live, work, and play. They impact...
by Madaline Spencer | Nov 13, 2024
Michael Hehir, MD, Professor of Neurology at the University of Vermont, discusses traditional versus novel therapies for myasthenia gravis (MG). MG is a chronic autoimmune neuromuscular disease characterized by weakness of the skeletal muscles. The...
by Madaline Spencer | Nov 12, 2024
Joshua Todd, PhD, National Institute of Neurological Disorders and Stroke at the NIH, discusses the clinical development of adeno-associated virus (AAV)-based gene therapies for neuromuscular disorders. The development of new genetic therapies is essential to...
by Madaline Spencer | Nov 11, 2024
Revital Lavi, MD, Pediatric Neurologist at the Sourasky Medical Center, discusses real-life outcome data with risdiplam for spinal muscular atrophy (SMA). The data was recently presented at the Annual Conference of the World Muscle Society (WMS 2024) held in Prague,...
by Madaline Spencer | Nov 8, 2024
Ulla Werlauff, PhD, The Danish Rehabilitation Centre for Neuromuscular Diseases, discusses the effect of medical treatment in non-ambulatory patients with spinal muscular atrophy (SMA). SMA is a group of genetic neuromuscular disorders due to mutations along...
