Rare Diseases Research: Clinical Trial for Niemann-Pick Type C
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Last updated 28 março 2025

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PDF] Diagnostic tests for Niemann-Pick disease type C (NP-C): A critical review.

Science Update: Biomarker for Niemann-Pick type C may be useful for diagnosing other class of rare disorders, NIH study suggests

Niemann-Pick disease type C: Video & Anatomy

Recent advances in the treatment of Niemann pick disease type C: A mini-review - ScienceDirect

Rare Diseases Research: Clinical Trial for Niemann-Pick Type C

TRIALS: A Fight for Children with Niemann-Pick Type C, a Fatal Genetic Disease

NIH teams with industry to develop treatments for Niemann-Pick Type C disease.

Parents of kids with Niemann Pick C advocate for adrabetadex
mRNA Treatment Rescues Niemann–Pick Disease Type C1 in Patient Fibroblasts
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