Casa niemann pick tipo c

Therapeutic intervention for Niemann–Pick type C disease must target

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Last updated 24 abril 2025

Therapeutic intervention for Niemann–Pick type C disease must target

Niemann-Pick type C: contemporary diagnosis and treatment of a classical disorder

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

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

IJMS, Free Full-Text

Niemann-Pick type C disease: molecular mechanisms and potential therapeutic approaches. - Abstract - Europe PMC

Understanding the phenotypic variability in Niemann-Pick disease type C (NPC): a need for precision medicine

Niemann–Pick type C disease: cellular pathology and pharmacotherapy - Wheeler - 2020 - Journal of Neurochemistry - Wiley Online Library

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

Tolerance of chronic HDACi treatment for neurological, visceral and lung Niemann-Pick Type C disease in mice

Proposed pathological cascade in Niemann-Pick disease type C (NPC)

Juvenile CLN3 disease is a lysosomal cholesterol storage disorder: similarities with Niemann-Pick type C disease - eBioMedicine

Niemann-Pick disease A or B in four pediatric patients and SMPD1 mutation carrier frequency in the Mexican population

Consensus clinical management guidelines for Niemann-Pick disease type C, Orphanet Journal of Rare Diseases

Recommendations on the diagnosis and management of Niemann-Pick disease type C - ScienceDirect

Correlation of age of onset and clinical severity in Niemann–Pick disease type C1 with lysosomal abnormalities and gene expression

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