by Madaline Spencer | Sep 29, 2025
The U.S. Food and Drug Administration (FDA) has approved Palsonify (paltusotine) for the first-line treatment of adults with acromegaly with inadequate response to surgery and/or for whom surgery is not an option. Acromegaly is a hormonal disorder that results from...
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...
