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...
by Madaline Spencer | Nov 5, 2024
Niklas Huntemann, University Hospital of Düsseldorf, discusses real-world experience with therapies targeting either the complement system (C5) or neonatal Fc receptor (FcRn) in patients with myasthenia gravis (MG). MG is a chronic autoimmune...
by Madaline Spencer | Nov 4, 2024
Michelle Mellion, MD, Chief Medical Officer at PepGen, discusses results from the CONNECT2-EDO51 study testing the safety and efficacy of PGN-EDO51 in people with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. DMD affects the muscles,...
by CheckRare Staff | Nov 1, 2024
Dr. Sujit Sheth, Chief of the Division of Pediatric Hematology/Oncology at Weill Cornell Medicine and an attending pediatrician at New York Presbyterian Hospital, in New York City discusses his recently published work in Blood Advances titled “Rates of severe...
