Records containing: Metachromatic Leukodystrophy
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.
Follow recorded terms in Drug Alerts for Android. Free includes unlimited local condition follows and an on-open recent-record watchlist. Plus adds no ads and personal organization tools.
All
FDA submission records
EMA medicine records
ClinicalTrials.gov record updates
ClinicalTrials.gov records with results posted
-
CLINICAL_TRIALS study record update · 2026-03-27and Safety of METAZYM (Recombinant Human Arylsulfatase A or rhASA) for the Treatment of Patients With Late Infantile MLD Who Had Previously Hematopoietic Stem Cell Transplantation
-
CLINICAL_TRIALS study record update · 2026-03-16A Single Arm, Open Label, Clinical Study of Cryopreserved Autologous CD34+ Cells Transduced With Lentiviral Vector Containing Human ARSA cDNA (OTL-200), for the Treatment of Early Onset Metachromatic Leukodystrophy (MLD)
-
CLINICAL_TRIALS study record update · 2026-03-02A Phase I/II, Open Labeled, Monocentric Study of Direct Intracranial Administration of a Replication Deficient Adeno-associated Virus Gene Transfer Vector Serotype rh.10 Expressing the Human ARSA cDNA to Children With Metachromatic Leukodystrophy.
-
CLINICAL_TRIALS study record update · 2026-01-07MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG
-
CLINICAL_TRIALS study record update · 2025-12-05A Phase I/II Clinical Trial of Hematopoietic Stem Cell Gene Therapy for the Treatment of Metachromatic Leukodystrophy
- Organize your saved records without ads with Plus.
-
CLINICAL_TRIALS study record update · 2025-10-23The Myelin Disorders Biorepository Project and Global Leukodystrophy Initiative Clinical Trials Network
-
CLINICAL_TRIALS study record update · 2025-09-24MATRIX - "Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia"
-
CLINICAL_TRIALS study record update · 2025-09-12ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
-
CLINICAL_TRIALS study record update · 2025-09-05An Open Label, Non-randomized Trial to Evaluate the Safety and Efficacy of a Single Infusion of OTL-200 in Patients With Late Juvenile (LJ) Metachromatic Leukodystrophy (MLD).
-
CLINICAL_TRIALS study record update · 2025-08-12Universal Genomic Newborn Screening in the Wallonia-Brussels Federation: Baby Detect
-
CLINICAL_TRIALS study record update · 2025-06-15LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
-
CLINICAL_TRIALS study record update · 2025-06-08A Systems-based Approach to Patient-focused Rare Disease Research and Product Development
-
CLINICAL_TRIALS study record update · 2025-04-04Early Check: A Collaborative Innovation to Facilitate Pre-Symptomatic Clinical Trials in Newborns
-
CLINICAL_TRIALS study record update · 2024-05-30The Natural History of Metachromatic Leukodystrophy
-
CLINICAL_TRIALS study record update · 2022-10-25A Single-Arm Study to Assess the Safety of Transplantation With Human Placental-Derived Stem-Cells Combined With Unrelated and Related Cord Blood in Subjects With Certain Malignant Hematologic Diseases and Non-Malignant Disorders
-
CLINICAL_TRIALS study record update · 2022-07-18Allogeneic Stem Cell Transplantation for the Treatment of Multiple Sclerosis (Compassionate Use)
-
CLINICAL_TRIALS study record update · 2022-05-31A Phase I/II Clinical Trial of Lentiviral Hematopoietic Stem Cell Gene Therapy for Treatment of Developed Metachromatic Leukodystrophy and Adrenoleukodystrophy
-
CLINICAL_TRIALS study record update · 2020-10-08Single Patient Expanded Access Protocol: Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
-
CLINICAL_TRIALS study record update · 2020-10-05Expanded Access Protocol for CaspaCIDe T Cells From An HLA-Partially Matched Related Donor After Negative Selection of TCR αβ+T Cells In Pediatric Patients Affected by Hematological and Other Disorders
-
CLINICAL_TRIALS study record update · 2019-07-11Treatment of High Risk, Inherited Lysosomal And Peroxisomal Disorders by Reduced Intensity Hematopoietic Stem Cell Transplantation
-
CLINICAL_TRIALS study record update · 2011-06-10Treatment of Lysosomal and Peroxisomal Inborn Errors of Metabolism by Bone Marrow Transplantation
-
CLINICAL_TRIALS study record update · 2011-03-21Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy