LEUKODYSTROPHY, GLOBOID CELL

Clinical trials for LEUKODYSTROPHY, GLOBOID CELL explained in plain language.

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Trials to join now! 1 Not yet finished but already full! 1

One-Shot gene therapy trial offers hope for devastating infant disease

Disease control Terminated

This study is testing a one-time gene therapy called PBKR03 for babies with early infantile Krabbe disease, a rare and severe neurological disorder. Researchers will give the therapy directly into the fluid around the brain to deliver a working copy of a missing gene. The main go…

Matched conditions: LEUKODYSTROPHY, GLOBOID CELL

Phase: PHASE1, PHASE2 • Sponsor: Gemma Biotherapeutics • Aim: Disease control

Last updated Mar 30, 2026 14:33 UTC

LEUKODYSTROPHY, GLOBOID CELL

Get alerted when new LEUKODYSTROPHY, GLOBOID CELL trials appear

One-Shot gene therapy trial offers hope for devastating infant disease