A hopeful therapy for Niemann-Pick C diseases - The Lancet
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Lysosomal storage diseases—the horizon expands
Full article: Current advancements in therapy for Niemann-Pick
Frontiers Emerging Concepts in Vector Development for Glial Gene
A Second Chance at Life”: Can Gene Therapies Beat Rare Disease
eBioMedicine, June 2023, Volume 92
Clinical applications of gene therapy for rare diseases: A review
Pharmaceuticals, Free Full-Text
Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological
Inhibition of Histone Deacetylases 1, 2, and 3 Enhances Clearance
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