Therapeutic intervention for Niemann–Pick type C disease must target
Por um escritor misterioso
Last updated 26 março 2025


Parents of kids with Niemann Pick C advocate for adrabetadex

Diagnostic workup and management of patients with suspected Niemann-Pick type C disease - Apostolos Papandreou, Paul Gissen, 2016

Progression of Niemann–Pick type C disease in mice and humans

Niemann-Pick Disease Type C Gene Therapy Gets Rare Pediatric Disease Designation

Niemann-Pick disease type C, Orphanet Journal of Rare Diseases

Psychiatric and Cognitive Symptoms Associated with Niemann-Pick Type C Disease: Neurobiology and Management

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

IJMS, Free Full-Text

Frontiers Adult-Onset Niemann–Pick Disease Type C: Rapid Treatment Initiation Advised but Early Diagnosis Remains Difficult

Niemann–Pick type C disease: cellular pathology and pharmacotherapy - Wheeler - 2020 - Journal of Neurochemistry - Wiley Online Library
Niemann Pick Disease Type C - Symptoms, Causes, Treatment

New murine Niemann-Pick type C models bearing a pseudoexon-generating mutation recapitulate the main neurobehavioural and molecular features of the disease

Main therapies and point of action in Niemann type C disease.
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