Current and Emerging Issues in Wilsons Disease
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X Linked Adrenoleukodystrophy:Clinical Presentation, Diagnosis, and Therapy
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Long-Term Effects of Antidarsagene Autotemcel for Metachromatic Leukodystrophy
NEJM 392:1609-1620, Fumagalli,F.,et al, 2025
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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Hematopoietic Stem - and Progenitor-Cell Gene Therapy for Hurler Syndrome
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Muscular Dystrophies
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Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
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Long-Term Safety and Tolerability of ProSavin, a Lentiviral Vector-Based Gene Therapy for Parkinsons Disease: A Dose Escalation, Open-Label, Phase 1/2 Trial
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Diagnosis and New Treatments in Muscular Dystrophies
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Diagnosis and Therapy in Neuromuscular Disorders: Diagnosis and New Treatments in Mitochondrial Diseases
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Neurovirological Methods and Their Applications
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