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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.
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Neurofilament light chain and glial fibrillary acidic protein levels in metachromatic leukodystrophy.
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Metachromatic leukodystrophy: A single-center longitudinal study of 45 patients.
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Clinical, Biochemical, and Molecular Characterization of Metachromatic Leukodystrophy Among Egyptian Pediatric Patients: Expansion of the ARSA Mutational Spectrum.
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Estimating the relative frequency of leukodystrophies and recommendations for carrier screening in the era of next-generation sequencing.
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Sulfatide levels correlate with severity of neuropathy in metachromatic leukodystrophy.
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