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.
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Niemann-Pick disease type C as a neurovisceral disease. Schematic
Niemann–Pick type C disease: cellular pathology and pharmacotherapy - Wheeler - 2020 - Journal of Neurochemistry - Wiley Online Library
New report gives national voice to patients, families affected by Niemann-Pick Type C, News, Notre Dame News
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