Hunter syndrome: Wisconsin boy receiving breakthrough treatment

Upcoming 2026 FDA Decisions Could Expand Treatment Options Across Oncology and Hepatitis B

Accessed July 7, 2026. 7. Ionis’ partner GSK announces bepirovirsen accepted for priority review and granted breakthrough therapy designatio

Teen Diagnosed with Rare Condition After Illness

7-year-old becomes Stanford's first proton therapy patient, showcasing breakthrough in cancer treatment accessibility

A tech breakthrough fits a specialized type of radiation treatment into standard treatment rooms, including at Stanford Medicine.

VNDA Stock Gains After-Hours As FDA Grants Rare Pediatric Disease Designation For Experimental Therapy

The designation covers VCA-894A, Vanda’s investigational treatment for Charcot-Marie-Tooth disease type 2S.

Roche ends 2 Huntington’s disease programs, including Ionis-partnered tominersen

Roche has halted development on two of its Huntington’s disease prospects, marking another blow for a patient community that has long awaite

New breakthroughs may bring us closer to gene-edited babies. Is that a good thing?

By Katie Hunt, CNN (CNN) — Pioneering gene-editing treatments are already in clinical use, saving lives and easing the pain of devastating g