Niemann-Pick B - RS - 5th infusion.
Por um escritor misterioso
Descrição
PDF) Novel first-dose adverse drug reactions during a phase I trial of olipudase alfa (recombinant human acid sphingomyelinase) in adults with Niemann–Pick disease type B (acid sphingomyelinase deficiency)
Interstitial lung disease in lysosomal storage disorders
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
Expanded access with intravenous hydroxypropyl-β-cyclodextrin to treat children and young adults with Niemann-Pick disease type C1: a case report analysis, Orphanet Journal of Rare Diseases
PDF) Reducing GBA2 Activity Ameliorates Neuropathology in Niemann-Pick Type C Mice
Frontiers Pre-clinical Mouse Models of Neurodegenerative Lysosomal Storage Diseases
(PDF) Niemann-Pick Disease: An Underdiagnosed Lysosomal Storage Disorder
Enhanced Delivery and Effects of Acid Sphingomyelinase by ICAM-1-Targeted Nanocarriers in Type B Niemann-Pick Disease Mice - ScienceDirect
Bilateral Cystic Bronchiectasis as Novel Phenotype of Niemann-Pick Disease Type B Successfully Treated With Double Lung Transplantation - CHEST
Frontiers Potential Disease-Modifying Effects of Lithium Carbonate in Niemann-Pick Disease, Type C1
Biomolecules, Free Full-Text
