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At a glance: the largest Niemann-Pick type C1 cohort with 602

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At a glance: the largest Niemann-Pick type C1 cohort with 602
Consensus clinical management guidelines for Niemann-Pick disease type C. - Abstract - Europe PMC
At a glance: the largest Niemann-Pick type C1 cohort with 602
Autophagy in liver diseases
At a glance: the largest Niemann-Pick type C1 cohort with 602
SEC Filing Sarepta Therapeutics, Inc.
At a glance: the largest Niemann-Pick type C1 cohort with 602
Revisiting the grammar of Tau aggregation and pathology formation: how new insights from brain pathology are shaping how we study and target Tauopathi - Chemical Society Reviews (RSC Publishing) DOI:10.1039/D1CS00127B
At a glance: the largest Niemann-Pick type C1 cohort with 602
Frontiers Review: Understanding Rare Genetic Diseases in Low Resource Regions Like Jammu and Kashmir – India
At a glance: the largest Niemann-Pick type C1 cohort with 602
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
At a glance: the largest Niemann-Pick type C1 cohort with 602
Deregulation of signalling in genetic conditions affecting the lysosomal metabolism of cholesterol and galactosyl-sphingolipids - ScienceDirect
At a glance: the largest Niemann-Pick type C1 cohort with 602
Clinical disease characteristics of patients with Niemann-Pick Disease Type C: findings from the International Niemann-Pick Disease Registry (INPDR), Orphanet Journal of Rare Diseases
At a glance: the largest Niemann-Pick type C1 cohort with 602
WORLDSymposium™ 2023
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