by Madaline Spencer | May 29, 2025
The U.S. Food and Drug Administration (FDA) has approved Yutrepia (treprostinil) inhalation powder for adults with pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease. PAH PAH is a rare condition affecting the...
by Madaline Spencer | May 29, 2025
James Griffin, sickle cell anemia patient advocate, gives advice to patients with sickle cell anemia and healthcare providers. Sickle cell anemia is a rare genetic disease in which the body produces red blood cells that are sickle-shaped. The sickle...
by Madaline Spencer | May 28, 2025
Kimberly LeBlanc, Genetic Counselor, Director of the Undiagnosed Diseases Network (UDN) Coordinating Center at Harvard Medical School, discusses approaching variants of uncertain significance in rare diseases. The UDN is a NIH-funded...
by Madaline Spencer | May 26, 2025
Annette Bakker, PhD, Chief Executive Officer of the Children’s Tumor Foundation, discusses repurposing shelved assets for rare diseases. The Children’s Tumor Foundation is an organization dedicated to developing treatments for patients with...
by Madaline Spencer | May 24, 2025
Alison Bateman-House, PhD, Assistant Professor Division of Medical Ethics at NYU Grossman School of Medicine, discusses ethical concerns in rare diseases and expanded access programs. Rare disease patients, caregivers, and healthcare practitioners often face...
