Yang P, Pardon LP, Ho AC, Lauer AK, et al. Safety and efficacy of ATSN-101 in patients with Leber congenital amaurosis
caused by biallelic mutations in GUCY2D: a phase 1/2, multicentre, open-label,
unilateral dose escalation study. Lancet 2024;404:962-970.
PMID: 39244273
![]() |
![]() |
![]() |