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

Por um escritor misterioso

Last updated 23 outubro 2024

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

Children with Niemann-Pick Type C (NPC), their parents and scientists seeking a treatment for this fatal genetic disease. WSJ follows this 6-year fight.

The legacy of finding a cure for Reno twins Addi and Cassie Hempel

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

Severely impaired CTL killing is a feature of the neurological disorder Niemann-Pick disease type C1 - ScienceDirect

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

Valley boy battling rare disease getting stronger

mRNA Treatment Rescues Niemann–Pick Disease Type C1 in Patient Fibroblasts

Niemann-Pick disease type C as a neurovisceral disease. Schematic

Greenwich family determined to defeat rare childhood disease

IJMS, Free Full-Text

Gene therapy shows promise for treating Niemann-Pick disease type C1

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