TRIALS: A Fight for Children with Niemann-Pick Type C, a Fatal Genetic Disease
Por um escritor misterioso
Descrição
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.
Frontiers Case Report: Be Aware of “New” Features of Niemann–Pick Disease: Insights From Two Pediatric Cases
Save Purnell Today
Your Impact - Ara Parseghian Medical Research Fund
delta – Columbus Children's Foundation
When Drug Development for Rare Disease Hit Setback, Parents Were Stung - WSJ
Niemann Pick Disease - an overview
Why Awareness Days Matter to Me as a Parent of a Kid With a Rare Disease
The legacy of finding a cure for Reno twins Addi and Cassie Hempel
Twins with rare, fatal disease at center of multimillion-dollar lawsuit
We know it works': Families, doctor fight move to discontinue medication
Two-Year-Old Kamryn Living with Niemann-Pick Disease Type C (NPC) - The Children's Inn at NIH
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