A hopeful therapy for Niemann-Pick C diseases - The Lancet
Por um escritor misterioso
Last updated 21 janeiro 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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