Metachromatic Leukodystrophy
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CLINICAL_TRIALS trial_result · 2026-04-21Clinical Trial Update: An Efficacy and Safety Study of HGT-1110 in Participants With Metachromatic Leukodystrophy [Phase 1, Phase 2]
This trial update reports on the Phase 1/2 efficacy and safety study of HGT-1110 in participants with Metachromatic Leukodystrophy (MLD). Th…
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CLINICAL_TRIALS trial_result · 2026-03-27Clinical Trial Update: Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell Transplantation [Phase 2]
This Phase 2 trial update reports on the efficacy of METAZYM for metachromatic leukodystrophy in patients who received hematopoietic stem ce…
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CLINICAL_TRIALS trial_result · 2026-03-16Clinical Trial Update: A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD) [Phase 2]
A Phase 2 clinical trial evaluating the safety and efficacy of cryopreserved OTL-200 for Metachromatic Leukodystrophy has completed.
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CLINICAL_TRIALS trial_result · 2026-03-02Clinical Trial Update: Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic Leukodystrophy [Phase 1, Phase 2]
This clinical trial update reports the completion of Phase 1/2 testing of an intracerebral gene therapy for children with early-onset Metach…
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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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EMA approval · 2025-12-16EMA Authorised: Libmeldy (atidarsagene autotemcel) — Orchard Therapeutics (Netherlands) B.V.
The European Medicines Agency authorised Libmeldy (atidarsagene autotemcel) from Orchard Therapeutics (Netherlands) B.V. for metachromatic l…
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CLINICAL_TRIALS trial_result · 2025-12-05Clinical Trial Update: Gene Therapy for Metachromatic Leukodystrophy (MLD) [Phase 1, Phase 2]
A gene therapy trial for Metachromatic Leukodystrophy (MLD) in Phase 1/2 has completed the study, sponsored by Orchard Therapeutics.
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CLINICAL_TRIALS trial_result · 2025-11-20Clinical Trial Update: Imaging Study of the White Matter Lesions in Children With Metachromatic Leucodystrophy [NA]
This document reports a completed imaging study evaluating white matter lesions in children with metachromatic leukodystrophy.
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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 trial_result · 2025-09-05Clinical Trial Update: OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD) [Phase 3]
The update describes a Phase 3 trial of OTL-200 for late juvenile Metachromatic Leukodystrophy and notes the study is currently not recruiti…
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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…
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CLINICAL_TRIALS trial_result · 2025-06-08Clinical Trial Update: The Natural History of Metachromatic Leukodystrophy Study (HOME Study)
The HOME natural history study for Metachromatic Leukodystrophy has completed.
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CLINICAL_TRIALS trial_result · 2024-06-06Clinical Trial Update: A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy [Phase 2]
Phase 2 study evaluating intrathecal SHP611 in children with Metachromatic Leukodystrophy; results have been posted and the trial is current…
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CLINICAL_TRIALS withdrawal · 2024-05-30Clinical Trial Update: The Natural History of Metachromatic Leukodystrophy
A clinical trial on the natural history of Metachromatic Leukodystrophy sponsored by the University of Pittsburgh has been withdrawn.
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CLINICAL_TRIALS trial_result · 2022-05-31Clinical Trial Update: Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy [Phase 1, Phase 2]
Clinical trial update for autologous hematopoietic stem cell gene therapy targeting Metachromatic Leukodystrophy and Adrenoleukodystrophy in…
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EMA approval · 2020-10-16New gene therapy to treat rare genetic disorder metachromatic leukodystrophy
EMA announces a new gene therapy to treat metachromatic leukodystrophy, a rare genetic disorder, marking a significant treatment option adva…
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CLINICAL_TRIALS trial_result · 2019-07-11Clinical Trial Update: HSCT for High Risk Inherited Inborn Errors [Phase 2]
A Phase 2 study updating hematopoietic stem cell transplantation for high-risk inherited metabolic disorders shows completed status with res…
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CLINICAL_TRIALS trial_result · 2018-10-15Clinical Trial Update: Multicenter Study of HGT-1110 Administered Intrathecally in Children With Metachromatic Leukodystrophy (MLD) [Phase 1, Phase 2]
Phase 1/2 multicenter trial of intrathecal HGT-1110 in children with metachromatic leukodystrophy (MLD) completed with results posted in 201…
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CLINICAL_TRIALS trial_result · 2015-08-13Clinical Trial Update: Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD) [Phase 1]
Phase 1 clinical trial update for Metazym in late infantile Metachromatic Leukodystrophy (MLD) has completed with results posted.
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CLINICAL_TRIALS trial_result · 2011-06-10Clinical Trial Update: Stem Cell Transplant for Inborn Errors of Metabolism [Phase 2, Phase 3]
Phase 2/3 clinical trial update on stem cell transplant for inborn errors of metabolism; trial completed with results posted in 2011.
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CLINICAL_TRIALS trial_result · 2011-03-21Clinical Trial Update: Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy [NA]
This document notes that a clinical trial evaluating warfarin as a treatment for metachromatic leukodystrophy has completed.