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

Gene therapy offers hope for kids with fatal batten disease

Disease control Completed

This study tested a one-time gene therapy for children with a rare, severe brain disease called variant late infantile Batten disease. The treatment delivers a working CLN6 gene into the spinal fluid to help slow the loss of motor and language skills. The trial included 13 childr…

Matched conditions: VARIANT LATE-INFANTILE NEURONAL CEROID LIPOFUSCINOSIS

Phase 1/2 • Sponsor: Emily de los Reyes • Aim: Disease control

Last updated Jun 27, 2026 07:55 UTC

Variant late-infantile neuronal ceroid lipofuscinosis

Get alerted when new Variant late-infantile neuronal ceroid lipofuscinosis trials appear

Gene therapy offers hope for kids with fatal batten disease