Experimental treatment for Niemann-Pick disease appears safe
Por um escritor misterioso
Descrição
An experimental drug has appeared to slow the progression of Niemann-Pick disease the drug 2-hydroxypropyl-beta-cyclodextrin is being tested
Why are people fighting over a promising treatment for a fatal childhood disease?, Science
Miglustat in Niemann-Pick disease type C patients: a review, Orphanet Journal of Rare Diseases
A Practical Chromogenic Procedure for the Detection of Homozygotes and Heterozygous Carriers of Niemann-Pick Disease
Orphazyme finds reduced Niemann-Pick disease progression in trial
Valley boy battling rare disease getting stronger
Dermatologic Manifestations of Niemann-Pick Disease: Background, Pathophysiology, Etiology
Treatment outcomes following continuous miglustat therapy in patients with Niemann-Pick disease Type C: a final report of the NPC Registry, Orphanet Journal of Rare Diseases
Niemann-Pick disease Information
Identification of Novel Biomarkers for Niemann–Pick Disease Using Gene Expression Analysis of Acid Sphingomyelinase Knockout Mice: Molecular Therapy
Experimental treatment for Niemann-Pick disease type C1 appears safe, effective
Families plead with FDA for drug to treat Niemann-Pick Disease Type C
Niemann–Pick Disease Type C
Zevra Therapeutics – NNPDF
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