PTC Therapeutics plans to request approval for its GT-AADC gene therapy, developed for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency, in the United States. Supported by positive data from clinical studies, the company expects to submit a biologics license application (BLA) with the U.S. Food and Drug Administration…
News
Recent Posts
- FDA unveils plan to speed personalized treatments for ultra-rare diseases
- Learning about rare disease through education and by sharing our own story
- Access to care for rare diseases still varies by state: NORD report card
- A long day of play reminds us why a child’s sleep schedule is important
- Digital clinical measures launched to support rare pediatric disease trials