LYSOSOMAL STORAGE DISEASES

Clinical trials for LYSOSOMAL STORAGE DISEASES explained in plain language.

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One-Time gene therapy aims to halt devastating childhood brain disease

Disease control Ongoing

This study is testing a single infusion of an experimental gene therapy called OTL-200 for children and teenagers with a rare, progressive brain disease called Late Juvenile Metachromatic Leukodystrophy (MLD). The therapy involves collecting a patient's own blood stem cells, addi…

Matched conditions: LYSOSOMAL STORAGE DISEASES

Phase: PHASE3 • Sponsor: Orchard Therapeutics • Aim: Disease control

Last updated Apr 03, 2026 14:43 UTC

LYSOSOMAL STORAGE DISEASES

Get alerted when new LYSOSOMAL STORAGE DISEASES trials appear

One-Time gene therapy aims to halt devastating childhood brain disease