TRIALS: A Fight for Children with Niemann-Pick Type C, a Fatal Genetic Disease
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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.
For a Rare Disease, Drug Trials Scramble for Patients - WSJ
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Gene therapy shows promise for treating Niemann-Pick disease type C1
Niemann–Pick disease, type C - Wikipedia
Niemann-Pick Disease, Type a disease: Malacards - Research Articles, Drugs, Genes, Clinical Trials
Rare Disease Video - Niemann-Pick Disease Types A & B - National Organization for Rare Disorders
PDF) Recent Advances in the Diagnosis and Treatment of Niemann-Pick Disease Type C in Children: A Guide to Early Diagnosis for the General Pediatrician
Frontiers Case Report: Be Aware of “New” Features of Niemann–Pick Disease: Insights From Two Pediatric Cases
TRIALS: A Fight for Children with Niemann-Pick Type C, a Fatal Genetic Disease
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