A hopeful therapy for Niemann-Pick C diseases - The Lancet
Por um escritor misterioso
Last updated 14 abril 2025


Investigation of 2-Hydroxypropyl-β-Cyclodextrin Treatment in a

The Lancet, 14 October 2017, Volume 390, Issue 10104, Pages

Investigation of 2-Hydroxypropyl-β-Cyclodextrin Treatment in a

Biomarker analysis of Niemann-Pick disease type C using

Marta, with Niemann Pick C, fights for a cure with gene therapy

Lysosomal storage diseases: Diagnostic confirmation and management

Understanding the phenotypic variability in Niemann-Pick disease

Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological

Neurological manifestations of lysosomal disorders and emerging

Investigation of 2-Hydroxypropyl-β-Cyclodextrin Treatment in a

PDF) Diagnostic workup and management of patients with suspected

Families plead with FDA for drug to treat Niemann-Pick Disease Type C

PDF) Identification of Niemann–Pick C1 (NPC1) disease biomarkers

Lysosomal storage disorders: from biology to the clinic with
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