by Madaline Spencer | Aug 25, 2025
Mohamed Ladha, President and General Manager for Recordati Rare Diseases North America, discusses the company’s rare disease initiatives. The U.S. branch of Recordati was established in 2013 with a focus on products to treat patients with rare inherited...
by Madaline Spencer | Aug 22, 2025
The U.S. Food and Drug Administration has approved Dawnzera (donidalorsen) for prophylaxis treatment to prevent attacks in patients ages 12 years and older with hereditary angioedema (HAE). HAE is a genetic condition characterized by recurrent episodes of severe...
by Madaline Spencer | Aug 22, 2025
Alan Krasner, MD, Chief Endocrinologist at Crinetics Pharmaceuticals, discusses data on the efficacy and safety of paltusotine for the treatment of acromegaly. Acromegaly is an endocrine disorder that results from the overproduction of growth hormone in...
by Madaline Spencer | Aug 20, 2025
Carla Nester, MD, University of Iowa Stead Family Children’s Hospital and lead investigator of the VALIANT study, discusses the impact of the recent approval of pegcetacoplan (Empaveli) for patients with C3 glomerulopathy (C3G) and immune complex...
by Madaline Spencer | Aug 19, 2025
Jamie Koprivnikar, MD, Hematologist Oncologist at Hackensack University Medical Center, discusses her clinical experience with iptacopan for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). PNH is an acquired disorder that leads to the...
