by Madaline Spencer | Aug 31, 2026
Eric Scheeff, PhD, Chief Scientific Officer at iDefine, discusses the treatment development of RNA amplifiers in patients with Kleefstra syndrome. Kleefstra syndrome is a rare disorder characterized by developmental delay and intellectual disability,...
by Madaline Spencer | Aug 28, 2026
Henry Kaminski, MD, Department of Neurology, and Linda Kusner, PhD, Department of Pharmacology & Physiology at George Washington University, discuss predicting response outcomes with proteomic and machine learning analyses in treating patients with myasthenia...
by Madaline Spencer | Aug 27, 2026
Tracey Dawson, PhD, Senior Vice President, US Therapeutic Area Head Neuroscience at Novartis, discusses the collaboration between Novartis, SMAshing My Limits, and Becoming rentABLE, striving to make travel accessible. Spinal muscular atrophy (SMA) is a...
by Madaline Spencer | Aug 26, 2026
The US Food and Drug Administration (FDA) has approved Imaavy (nipocalimab) for the treatment of patients ages 12 years and older with warm autoimmune hemolytic anemia (wAIHA) who are currently or previously treated with corticosteroids. wAIHA is a rare hematologic...
by Madaline Spencer | Aug 24, 2026
Gaia Coppock, MD, Nephrologist and Associate Professor of Clinical Medicine at the University of Pennsylvania, discusses patient experiences with IgA nephropathy (IgAN). IgAN is characterized by the accumulation of IgA antibodies in the kidneys. In the...
