Research Summary: January 2022 Research summary of recent leukodystrophy research and clinical trials, includes titles and direct links to websites and articles.
ALD Molecular Biomarkers for Adrenoleukodystrophy: An Unmet Need
Traditional Chinese medicine for gait disturbance in adrenoleukodystrophy: A case report and review of literature
Vitamin D Status & Latitude Predict Brain Lesions in Adrenoleukodystrophy
Adrenoleukodystrophy National Registry Study
Alexander Disease Alexander disease evolution over time: data from an Italian cohort of pediatric-onset patients
Alexander disease: models, mechanisms, and medicine
Type II Alexander disease with fragile X mental retardation 1 gene mutation
Metabolic Enzyme Alterations and Astrocyte Dysfunction in a Murine Model of Alexander Disease With Severe Reactive Gliosis
Antisense therapy in a rat model of Alexander disease reverses GFAP pathology, white matter deficits, and motor impairment
Teaching NeuroImage: Dorsal Medullary Lesions in Juvenile-Onset Alexander Disease
CADASIL A novel heterozygous HTRA1 mutation in an Asian family with CADASIL-like disease
Canavan Disease Passage Bio Announces Pipeline Expansion and Clinical Program Update
Myrtelle Announces Exclusive Worldwide Gene Therapy Licensing Agreement for Canavan Disease
Selective screening for inborn errors of metabolism by tandem mass spectrometry at Sohag University Hospital, Egypt
Cockayne Syndrome Four-stranded DNA structure interactions play role in ageing syndrome
GM2 gangliosidosis and Giant Axonal Neuropathy (GAN) Taysha Gene Therapies Announces January 2022 Virtual Investor Events to Review Preliminary Clinical Data for TSHA-101 in GM2 Gangliosidosis and High Dose Cohort and Long-Term Clinical Data for TSHA-120 in Giant Axonal Neuropathy
Krabbe Disease Gain Therapeutics Progresses Krabbe Disease Program and Provides Scientific Update
Leigh Syndrome Diagnosing mitochondrial conditions: a WGS breakthrough
MLD A Mutation-Agnostic Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy
