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Ocular manifestations of Neuronal Ceroid Lipofuscinosis (CLN) Type 2 research hub

1 indexed studies · 0 currently recruiting

authorisedPhase I and Phase II (Integrated)- First administration to humans
A First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Tolerability of Gene Therapy with TTX-381 for the Ocular Manifestations Associated with Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease
This study is testing a new gene therapy, TTX-381, for children with CLN2 disease. It's the first time this treatment has been given to people. Researchers want to see if it's safe and helps with eye problems linked to the disease.
Germany