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Sponsored AI AM CLAUDE, A Beginner's Guide: Learn the basics of working with AI AI AM CLAUDE, A Beginner's Guide: Learn the basics of working with AI

Author

D. Drozdov

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Review Open access Oct 2026

A Systematic Review of Metachromatic Leukodystrophy Newborn Screening: Transitioning from Pilot Studies to the Real World

Metachromatic leukodystrophy (MLD) is a rare lysosomal storage disorder causing progressive neurodegeneration through central and peripheral demyelination, leading to premature death. Recent advances in ex vivo lentiviral gene therapy have developed a potentially curative treatment approach for pre-symptomatic infants...

Samuel Waser, Simone Kistler, L. Adang et al. · 0 citations

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