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...

Linerixibat Reduces Itching in Patients with PBC

by | Jun 13, 2025

Robert Mitchell-Thain, chair of the PBC Foundation, discusses how linerixibat reduces itching in patients with primary biliary cholangitis (PBC).     PBC is a chronic, progressive autoimmune liver disease in which the bile ducts become inflamed and damaged....