A hopeful therapy for Niemann-Pick C diseases - The Lancet
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Descrição
Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological
Cyclodextrin Shows Promise in Niemann-Pick Type C Disease
Understanding the phenotypic variability in Niemann-Pick disease
Pharmaceuticals, Free Full-Text
Miglustat for treatment of Niemann-Pick C disease: a randomised
A hopeful therapy for Niemann-Pick C diseases - The Lancet
Decreased neural stem cell proliferation and olfaction in mouse
Improved systemic AAV gene therapy with a neurotrophic capsid in
Diagnostic approach in adult-onset neurometabolic diseases
Effects of miglustat therapy on neurological disorder and survival
IJMS, Free Full-Text
Identification of Niemann-Pick C1 disease biomarkers through
Clinical applications of gene therapy for rare diseases: A review
New perspectives for preventing hepatitis C virus liver graft
Therapeutic intervention for Niemann–Pick type C disease must
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