by Madaline Spencer | Sep 29, 2025
Jamas LaFreniere, Founder of Sophie’s Hope Foundation and CURE GSD1b, and Blair Stone-Schneider, Executive Director of Sophie’s Hope Foundation, discuss their patient advocacy organization and starting a natural history study for glycogen storage disease type 1b...
by Madaline Spencer | Sep 26, 2025
Phioanh Leia Nghiemphu, MD, Professor of Clinical Neurology at University of California Los Angeles, discusses the use of mirdametinib for neurofibromatosis type 1 with symptomatic plexiform neurofibromas (NF1-PN). NF1 is a genetic condition that affects...
by Madaline Spencer | Sep 25, 2025
Geoffrey Liu, MD, Senior Scientist at the Princess Margaret Cancer Centre at the University of Toronto, discusses new data on the treatment of ROS1-positive non-small cell lung cancer (NSCLC) with taletrectinib. ROS1-positive NSCLC is a rare and...
by Madaline Spencer | Sep 24, 2025
Scott Baver, PhD, Vice President of Medical Affairs at ITF Therapeutics, discusses long-term safety and efficacy data on givinostat for patients with Duchenne muscular dystrophy (DMD). DMD is a rare neuromuscular disorder characterized by progressive...
by Madaline Spencer | Sep 23, 2025
The U.S. Food and Drug Administration (FDA) has granted accelerated approval to Forzinity (elamipretide HCI) for the treatment of patients with Barth syndrome. The treatment is indicated to improve muscle strength in adult and pediatric patients weighing at least 30...
