VARIANT LATE-INFANTILE NEURONAL CEROID LIPOFUSCINOSIS

Clinical trials for VARIANT LATE-INFANTILE NEURONAL CEROID LIPOFUSCINOSIS explained in plain language.

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Completed 1

One-Shot gene therapy tested to halt fatal childhood brain disease

Disease control Completed

This study tested a one-time gene therapy injection for children with a rare, fatal genetic brain disorder called CLN6 Batten disease. The therapy aimed to deliver a working copy of the faulty CLN6 gene directly to the nervous system to slow or stop disease progression. Researche…

Matched conditions: VARIANT LATE-INFANTILE NEURONAL CEROID LIPOFUSCINOSIS

Phase: PHASE1, PHASE2 • Sponsor: Emily de los Reyes • Aim: Disease control

Last updated Mar 27, 2026 12:40 UTC

VARIANT LATE-INFANTILE NEURONAL CEROID LIPOFUSCINOSIS

Get alerted when new VARIANT LATE-INFANTILE NEURONAL CEROID LIPOFUSCINOSIS trials appear

One-Shot gene therapy tested to halt fatal childhood brain disease