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.
Experimental treatment helps toddler with rare genetic disease - CBS News
Niemann–Pick disease, type C - Wikipedia
TRIALS: A Fight for Children with Niemann-Pick Type C, a Fatal Genetic Disease
Rare Disease to Inform Ebola Susceptibility?
Consensus clinical management guidelines for Niemann-Pick disease type C, Orphanet Journal of Rare Diseases
Rapid Diagnosis of 83 Patients with Niemann Pick Type C Disease and Related Cholesterol Transport Disorders by Cholestantriol Screening - eBioMedicine
For a Rare Disease, Drug Trials Scramble for Patients - WSJ
Ohio 2-year-old battling rare Niemann-Pick disease, dubbed 'childhood Alzheimer's
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
Rare Disease to Inform Ebola Susceptibility?
Two-Year-Old Kamryn Living with Niemann-Pick Disease Type C (NPC) - The Children's Inn at NIH
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