Niemann-Pick B - RS - 5th infusion.
Por um escritor misterioso
Last updated 29 maio 2024
Enhanced Delivery and Effects of Acid Sphingomyelinase by ICAM-1-Targeted Nanocarriers in Type B Niemann-Pick Disease Mice - ScienceDirect
JCM, Free Full-Text
Lysosomal storage diseases: Diagnostic confirmation and management of presymptomatic individuals
IJMS, Free Full-Text
Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1–2 trial - The Lancet
PDF) A Prospective, Cross-sectional Survey Study of the Natural History of Niemann-Pick Disease Type B
WO2022026410A2 - Compositions and methods for the treatment of niemann-pick type c1 disease - Google Patents
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
Frontiers Expert opinion on patient journey, diagnosis and clinical monitoring in acid sphingomyelinase deficiency in Turkey: a pediatric metabolic disease specialist's perspective
IJMS, Free Full-Text
Lysosomal storage diseases: Diagnostic confirmation and management of presymptomatic individuals
Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1–2 trial - The Lancet
Lysosomal Storage Disease disease: Malacards - Research Articles, Drugs, Genes, Clinical Trials
Advances in therapies for neurological lysosomal storage disorders - Ellison - 2023 - Journal of Inherited Metabolic Disease - Wiley Online Library
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