FDA Approves Gene Therapy For Rare Disorder After Rejecting It 2 Years Ago

FDA Approves Gene Therapy For Rare Disorder After Rejecting It 2 Years Ago The therapy marketed as Kresladi was initially rejected in 2024 because of manufacturing concerns. It treats leukocyte adhesion deficiency type 1 (LAD-1), an ultra-rare disease — affecting about one in a million — that makes children vulnerable to life-threatening infections and can lead to death. https://kffhealthnews.org/morning-breakout/fda-approves-gene-therapy-for-rare-disorder-after-rejecting-it-2-years-ago/