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...
by Madaline Spencer | Nov 7, 2024
Natalie Goedeker, Nurse Practitioner at Washington University School of Medicine in St. Louis, MO, discusses rapid initiation of risdiplam in newborns with spinal muscular atrophy (SMA). SMA is a group of genetic neuromuscular disorders due to mutations along...
by Madaline Spencer | Nov 6, 2024
Teresa Gidaro, Director of Clinical Development for NMD Pharma, discusses the SYNAPSE-CMT clinical trial testing NMD670 in the treatment of Charcot-Marie-Tooth (CMT) disease. CMT disease is a type of peripheral neuropathy, a condition affecting the transmission...
