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Last Updated: 01/20/2023

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Found 25 publications

Treatment of cerebral adrenoleukodystrophy: allogeneic transplantation and lentiviral gene therapy.

Condition: Cerebral Adrenoleukodystrophy (ALD)
Journal: Expert opinion on biological therapy
Treatment Used: Allogenic Transplantation and Lentiviral Gene Therapy
Number of Patients: 0
Published: September 15, 2022

The safety and efficacy of umbilical cord blood mononuclear cells in individuals with spastic cerebral palsy: a randomized double-blind sham-controlled clinical trial.

Condition: Spastic Cerebral Palsy (CP)
Journal: BMC neurology
Treatment Used: Umbilical Cord Blood Mononuclear Cells (UCB-MNC)
Number of Patients: 72
Published: March 30, 2022

Mucopolysaccharidosis-Plus Syndrome, a Rapidly Progressive Disease: Favorable Impact of a Very Prolonged Steroid Treatment on the Clinical Course in a Child.

Condition: Mucopolysaccharidosis-Plus Syndrome (MPS-PS)
Journal: Genes
Treatment Used: Steroids
Number of Patients: 1
Published: March 25, 2022

Hematopoietic Stem Cell Transplantation with Mesenchymal Stromal Cells in Children with Metachromatic Leukodystrophy.

Condition: Children with Metachromatic Leukodystrophy (MLD)
Journal: Stem cells and development
Treatment Used: Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) with Mesenchymal Stromal Cells (MSCs)
Number of Patients: 10
Published: March 24, 2022

Ketamine as an Adjunct for Treatment of Methotrexate-induced Neurotoxicity.

Condition: Methotrexate-induced Neurotoxicity
Journal: Journal of pediatric hematology/oncology
Treatment Used: Ketamine
Published: February 24, 2022

Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access.

Condition: Early-Onset Metachromatic Leukodystrophy
Journal: Lancet (London, England)
Treatment Used: Lentiviral Hematopoietic Stem-Cell Gene Therapy
Number of Patients: 31
Published: January 23, 2022

Human iPSC-Derived Astrocytes: A Powerful Tool to Study Primary Astrocyte Dysfunction in the Pathogenesis of Rare Leukodystrophies.

Journal: International journal of molecular sciences
Published: January 11, 2022

Four New Cases of Hypomyelinating Leukodystrophy Associated with the UFM1 c.-155_-153delTCA Founder Mutation in Pediatric Patients of Roma Descent in Hungary.

Journal: Genes
Published: September 28, 2021

Leukodystrophies in Children: Diagnosis, Care, and Treatment.

Journal: Pediatrics
Published: August 24, 2021

Clinical and imaging outcomes after intrathecal injection of umbilical cord tissue mesenchymal stem cells in cerebral palsy: a randomized double-blind sham-controlled clinical trial.

Condition: Cerebral Palsy
Journal: Stem cell research & therapy
Treatment Used: Intrathecal Injection of Umbilical Cord Tissue Mesenchymal Stem Cells
Number of Patients: 36
Published: August 07, 2021

The Role of White Matter Dysfunction and Leukoencephalopathy/Leukodystrophy Genes in the Aetiology of Frontotemporal Dementias: Implications for Novel Approaches to Therapeutics.

Journal: International journal of molecular sciences
Published: April 03, 2021

Genetic testing of leukodystrophies unraveling extensive heterogeneity in a large cohort and report of five common diseases and 38 novel variants.

Journal: Scientific reports
Published: February 06, 2021
Showing 1-12 of 25

Last Updated: 01/20/2023