TRIALS: A Fight for Children with Niemann-Pick Type C, a Fatal Genetic Disease
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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 complexity of a monogenic neurodegenerative disease: More than two decades of therapeutic driven research into Niemann-Pick type C disease - ScienceDirect
Deaf or Death? In Drug Trial, Parents Weigh Life vs. Hearing Loss - WSJ
Niemann-Pick disease type C, Orphanet Journal of Rare Diseases
Parents of kids with Niemann Pick C advocate for adrabetadex
Niemann-Pick Disease, Type a disease: Malacards - Research Articles, Drugs, Genes, Clinical Trials
Your Impact - Ara Parseghian Medical Research Fund
Morbidity and mortality in type B Niemann–Pick disease
Niemann Pick C Awareness Group
Two-Year-Old Kamryn Living with Niemann-Pick Disease Type C (NPC) - The Children's Inn at NIH
We know it works': Families, doctor fight move to discontinue medication
TRIALS: A Fight for Children with Niemann-Pick Type C, a Fatal Genetic Disease
Penn Vet Researchers Identify Effective Treatment for Niemann Pick Type C
