FDA Approves Garadacimab for Hereditary Angioedema

by | Jun 18, 2025

The U.S. Food and Drug Administration (FDA) has approved Andembry (garadacimab) for the treatment of hereditary angioedema (HAE) in patients ages 12 years and older.   Hereditary Angioedema HAE is a rare condition characterized by recurrent episodes of severe...

Evolving Policy Landscapes for Rare Disease Access

by | Jun 17, 2025

Deb Jennings, Head of North America Patient Services Operations at Kyowa Kirin, discusses evolving policy landscapes for rare disease access.     In a panel discussion at the 2025 World Orphan Drug Congress, Ms. Jennings explained the volatility of the...

Patient Experience With Stevens-Johnson Syndrome

by | Jun 16, 2025

Katie Niemeyer, patient advocate, discusses her experience with Stevens-Johnson syndrome/toxic epidermal necrolysis (SJS/TEN).     SJS/TEN is a very severe reaction that causes skin tissue to die and detach. SJS and TEN previously were thought to be separate...

FDA Approves Taletrectinib for Rare Form of Lung Cancer

by | Jun 13, 2025

The U.S. Food and Drug Administration (FDA) has approved taletrectinib for the treatment of adults with locally advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC). ROS1-positive NSCLC is a rare and aggressive lung cancer that typically occurs in...