Metachromatic Leukodystrophy
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS research · 2026-02-09Clinical Trial Update: Longitudinal Study of Neurodegenerative Disorders
This clinical trial update describes a longitudinal study of several neurodegenerative and lysosomal storage disorders. The study is current…
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CLINICAL_TRIALS research · 2025-10-23Clinical Trial Update: The Myelin Disorders Biorepository Project
This clinical trials update describes the Myelin Disorders Biorepository Project (NCT03047369) from Children’s Hospital of Philadelphia, cur…
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CLINICAL_TRIALS research · 2025-09-24Clinical Trial Update: Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia
A recruiting clinical trial from Assistance Publique - Hôpitaux de Paris is examining macrophage activation patterns in four leukodystrophie…
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CLINICAL_TRIALS research · 2025-09-12Clinical Trial Update: ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
This clinical trial update describes ScreenPlus, a comprehensive, multi-disorder newborn screening program that is currently enrolling by in…
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CLINICAL_TRIALS research · 2025-09-08Clinical Trial Update: UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells [Phase 1]
Phase 1 trial evaluating intrathecal umbilical cord blood-derived oligodendrocyte-like cells for several inherited metabolic brain diseases;…
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CLINICAL_TRIALS research · 2025-07-01Clinical Trial Update: Lentiviral Hematopoietic Stem Cell Gene Therapy for MLD [NA]
Clinical trial update shows recruitment is ongoing for a lentiviral hematopoietic stem cell gene therapy to treat Metachromatic Leukodystrop…
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CLINICAL_TRIALS research · 2025-06-26Clinical Trial Update: Direct Lentiviral Injection Gene Therapy for MLD [NA]
This entry notes that Shenzhen Geno-Immune Medical Institute is recruiting for a direct lentiviral injection gene therapy trial for Metachro…