Records containing: Metachromatic Leukodystrophy (MLD)
This is the wording recorded or indexed by the source. An agency index term is not necessarily an authorised indication. Co-listed drug names are not linked to this term as a treatment relationship.
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CLINICAL_TRIALS study record update · 2026-04-21An Open-Label Extension of Study HGT-MLD-070 Evaluating Long Term Safety and Efficacy of Intrathecal Administration of HGT-1110 in Patients With Metachromatic Leukodystrophy
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CLINICAL_TRIALS study record update · 2025-07-01Lentiviral Vector-modified Autologous Hematopoietic Stem Cells for Metachromatic Leukodystrophy (MLD)
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CLINICAL_TRIALS study record update · 2025-06-26Direct Lentiviral TYF-ARSA Injection Gene Therapy for Metachromatic Leukodystrophy (MLD)
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CLINICAL_TRIALS study record update · 2025-05-29Coordination of Rare Diseases at Sanford
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CLINICAL_TRIALS study record update · 2024-06-06A Global, Multicenter, Single-arm, Matched External Control Study of Intrathecal SHP611 in Subjects With Late Infantile Metachromatic Leukodystrophy
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CLINICAL_TRIALS study record update · 2024-01-12Characterization of Metachromatic Leukodystrophy (MLD) in Pediatric Population in Spain: Epidemiological, Clinical, Diagnostic, Therapeutic, and Socioeconomic Aspects
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CLINICAL_TRIALS study record update · 2023-04-12Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
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CLINICAL_TRIALS study record update · 2021-03-17Natural History Study of Children With Metachromatic Leukodystrophy
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CLINICAL_TRIALS study record update · 2018-10-15A Phase I/II Multicenter Open-label Dose Escalation Study of HGT-1110 Administered Intrathecally in Children With Metachromatic Leukodystrophy
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CLINICAL_TRIALS study record update · 2017-05-12Allogeneic Hematopoietic Stem Cell Transplantation for Standard Risk Inherited Metabolic Disorders
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CLINICAL_TRIALS study record update · 2015-08-13A Single Center, Open-label, Non-randomized, Uncontrolled, Multiple-dose, Dose Escalation Study of the Safety, Pharmacokinetics and Efficacy of Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)