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


A first therapy for Niemann-Pick C - The Lancet Neurology

Pharmaceuticals, Free Full-Text

Central nervous system therapy for lysosomal storage disorders in

Neurological manifestations of lysosomal disorders and emerging

Miglustat for treatment of Niemann-Pick C disease: a randomised

IJMS, Free Full-Text

PDF) Diagnostic workup and management of patients with suspected

Identification of Niemann-Pick C1 disease biomarkers through

Experimental treatment for Niemann-Pick disease type C1 appears

Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological

A hopeful therapy for Niemann-Pick C diseases,The Lancet - X-MOL

Understanding the phenotypic variability in Niemann-Pick disease

Lysosomal storage diseases: Diagnostic confirmation and management

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