Ongoing retinal degeneration despite intraventricular enzyme replacement therapy with cerliponase alfa in late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2 disease).
Simon DulzC SchweringJan WildnerChristoph SpartalisFrank SchuettaufUdo BartschEva WibbelerMiriam NickelMartin Stephan SpitzerYevgeniya AtiskovaAngela SchulzPublished in: The British journal of ophthalmology (2022)
NCT04613089.