1. Verhaart IEC, Aartsma-Rus A. Therapeutic developments for Duchenne muscular dystrophy. Nat Rev Neurol. 2019;15(7):373–86.
2. Messina S, Vita GL. Clinical management of Duchenne muscular dystrophy: the state of the art. Neurol Sci. 2018;39(11):1837–45.
3. Sarepta Therapeutics. Sarepta Therapeutics announces FDA approval of Vyondys 53™ (golodirsen) injection for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 53 [media release]. 12 Dec 2019.
4. Sarepta Therapeutics. Sarepta Therapeutics receives Complete Response Letter from the US Food and Drug administration for golodirsen new drug application [media release]. 19 Aug 2019.
5. US Food & Drug Administration. FDA grants accelerated approval to first targeted treatment for rare Duchenne muscular dystrophy mutation [media release]. Dec 12 2019.