Kebilidi, AADC deficiency gene therapy, now approved by FDA The U.S. Food and Drug Administration has approved PTC Therapeutics' one-time gene therapy, called Kebilidi, for people with AADC deficiency.

Gene therapy Kebilidi now approved in the US to treat those with AADC deficiency. aadcnews.com/news/kebilid...

#ptctherapeutics #kebilidi #aadc #rarediseases