#rare diseases

2 articles tagged with "rare diseases"

FDA Approves Denali Therapeutics Drug for Hunter Syndrome

A significant decision by the FDA following previous rejections of rare disease treatments.

Briefing: GOP Senator Investigates FDA Over Rare Disease Drug Rejections

Strategic angle: Sen. Ron Johnson raises concerns about the FDA's handling of treatments for rare diseases after discussions with Commissioner Marty Makary.

Editorial Staff about 1 month ago