METACHROMATIC LEUKODYSTROPHY (MLD)
Clinical trials for METACHROMATIC LEUKODYSTROPHY (MLD) explained in plain language.
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Spinal fluid injections aim to preserve movement in devastating childhood brain disease
Disease control OngoingThis study is testing whether an experimental drug called SHP611, injected into the spinal fluid, can help children with a severe genetic brain disease called MLD keep their ability to walk and move for longer. The trial involves about 36 children who will receive the injections …
Matched conditions: METACHROMATIC LEUKODYSTROPHY (MLD)
Phase: PHASE2 • Sponsor: Shire • Aim: Disease control
Last updated Apr 01, 2026 14:42 UTC
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Extended trial tests potential treatment for rare childhood brain disease
Disease control OngoingThis study continues testing an experimental treatment called HGT-1110 in children with metachromatic leukodystrophy (MLD), a rare and serious genetic brain disease. It follows 24 children who participated in an earlier trial, giving them regular spinal injections of the treatmen…
Matched conditions: METACHROMATIC LEUKODYSTROPHY (MLD)
Phase: PHASE1, PHASE2 • Sponsor: Shire • Aim: Disease control
Last updated Mar 23, 2026 15:15 UTC