Niemann-Pick B - RS - 5th infusion.
Por um escritor misterioso
Last updated 02 abril 2025

Promoter considerations in the design of lentiviral vectors for use in treating lysosomal storage diseases: Molecular Therapy - Methods & Clinical Development

Biomolecules, Free Full-Text

PDF) Metabolic improvement in patients with Niemann-Pick disease following intravenous trehalose administration: An untargeted pharmacometabolomic study

Frontiers Pre-clinical Mouse Models of Neurodegenerative Lysosomal Storage Diseases

Frontiers Expert opinion on patient journey, diagnosis and clinical monitoring in acid sphingomyelinase deficiency in Turkey: a pediatric metabolic disease specialist's perspective

Enhanced Delivery and Effects of Acid Sphingomyelinase by ICAM-1-Targeted Nanocarriers in Type B Niemann-Pick Disease Mice - ScienceDirect

Familial Hypercholesterolemia: From Clinical Suspicion to Novel Treatments

Long-term administration of intravenous Trappsol® Cyclo™ (HP-β-CD) results in clinical benefits and stabilization or slowing of disease progression in patients with Niemann-Pick disease type C1: Results of an international 48-week Phase I/II

Splenomegaly, hypersplenism, and hereditary disorders with splenomegaly

Lysosomal storage diseases: Diagnostic confirmation and management of presymptomatic individuals - Genetics in Medicine

Natural history and management of liver dysfunction in lysosomal storage disorders

Niemann-Pick C1 expression is not regulated by the amount of cholesterol flowing through cells in the mouse - ScienceDirect

WO2019130230A1 - Heterocyclic amides as kinase inhibitors - Google Patents

Dose-dependent and disease-modifying effects of striatal infusion of cholesterol in Huntington's disease
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