First in man study of intravitreal tripeptidyl peptidase 1 for CLN2 retinopathy.
James WawrzynskiAna Rodriguez MartinezDorothy Ann ThompsonDipak RamRichard BowmanRebecca WhiteleyChin GanLouise HardingAmanda MortensenPhilippa MillsPaul GissenRobert H HendersonPublished in: Eye (London, England) (2023)
Intravitreal rhTPP1 appears to be a safe and effective treatment for CLN2 related retinopathy however commencement of treatment early in the course of disease is more likely to be efficacious.