1. O. of the Commissioner (2019). FDA approves innovative gene therapy to treat pediatric patients with spinal muscular atrophy, a rare disease and leading genetic cause of infant mortality. FDA. https://www.fda.gov/news-events/press-announcements/fda-approves-innovative-gene-therapy-treat-pediatric-patients-spinal-muscular-atrophy-rare-disease.
2. O. of the Commissioner (2017). Press Announcements - FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss. FDA. https://www.fda.gov/newsevents/newsroom/pressannouncements/ucm589467.htm.
3. Spark Therapeutics LUXTURNA package insert. 1–16.
4. AveXis (2019). ZOLGENSMA® (onasemnogene abeparvovec-xioi) suspension for intravenous infusion [package insert]. Bannockburn (IL): AveXis Inc; 2019 May. Available from: https://www.fda.gov/media/126109/download. 1–14.
5. Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03333590.