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1st January 2022

Antisense therapy in a rat model of Alexander disease reverses GFAP pathology, white matter deficits, and motor impairment

Alexander Disease

1st January 2022

Metabolic Enzyme Alterations and Astrocyte Dysfunction in a Murine Model of Alexander Disease With Severe Reactive Gliosis

Alexander Disease

1st January 2022

Type II Alexander disease with fragile X mental retardation 1 gene mutation

Alexander Disease

1st January 2022

Alexander disease: models, mechanisms, and medicine

Alexander Disease

1st January 2022

Adrenoleukodystrophy National Registry Study

Adrenoleukodystrophy (ALD)

1st January 2022

Vitamin D Status & Latitude Predict Brain Lesions in Adrenoleukodystrophy

Adrenoleukodystrophy (ALD)

1st January 2022

Traditional Chinese medicine for gait disturbance in adrenoleukodystrophy: A case report and review of literature

Adrenoleukodystrophy (ALD)

1st January 2022

Molecular Biomarkers for Adrenoleukodystrophy: An Unmet Need

Adrenoleukodystrophy (ALD)

1st January 2022

Research Summary: January 2022

Research summary of recent leukodystrophy research and clinical trials, includes article summaries and direct links to websites and articles.

1st December 2021

Clinical trial using gene therapies for neurodegenerative diseases reports positive interim data

GM1 gangliosidosis

1st December 2021

Aldehyde-driven transcriptional stress triggers an anorexic DNA damage response

Cockayne Syndrome (CS)

1st December 2021

BridgeBio Pharma Announces Dosing of First Patient in Phase 1/2 Trial of Investigational Gene Therapy for Canavan Disease

Canavan Disease

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