Vc Thoughts, Health, Dna, Rna, Healthcare.
commercially approved for rare diseases that lack available treatments, such as Leber Congenital Amaurosis (Luxturna — Spark Therapeutics/Roche), Hemophilia A (Roctavian — BioMarin), and Duchenne Muscular Dystrophy (Elevidys — Sarepta Therapeutics). The approval for Elevidys shows that targeting