Experimental treatment for Niemann-Pick disease appears safe
Descrição
An experimental drug has appeared to slow the progression of Niemann-Pick disease the drug 2-hydroxypropyl-beta-cyclodextrin is being tested
Improved systemic AAV gene therapy with a neurotrophic capsid in Niemann–Pick disease type C1 mice
Molecular Mind Games: The Medicinal Action of Cyclodextrins in Neurodegenerative Diseases
Niemann-Pick Disease Concise Medical Knowledge
Consensus clinical management guidelines for Niemann-Pick disease type C, Orphanet Journal of Rare Diseases
IJMS, Free Full-Text
Experimental treatment for Niemann-Pick disease type C1 appears safe, effective
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
Niemann-Pick disease type C
Families plead with FDA for drug to treat Niemann-Pick Disease Type C
Enhanced Delivery and Effects of Acid Sphingomyelinase by ICAM-1-Targeted Nanocarriers in Type B Niemann-Pick Disease Mice - ScienceDirect
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A new treatment can delay effects of Neimann-Pick disease in children
INPDA International Niemann-Pick Disease Alliance
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