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

At a glance: the largest Niemann-Pick type C1 cohort with 602 patients diagnosed over 15 years

TFEB and TFE3 control glucose homeostasis by regulating insulin gene expression

Characteristics of the cohort of 602 NPC1 patients.

Spot on biotechnology science new edition 2016 by BIO.NRW Cluster Biotechnology NRW - Issuu

Familial ALS in Germany: origin of the R115G SOD1 mutation by a founder effect

Clinical disease characteristics of patients with Niemann-Pick Disease Type C: findings from the International Niemann-Pick Disease Registry (INPDR). - Abstract - Europe PMC

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

Autophagy in liver diseases

Staphylococcus aureus Infections: Epidemiology, Pathophysiology, Clinical Manifestations, and Management

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

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