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
Familial Hypercholesterolemia: From Clinical Suspicion to Novel Treatments
Experimental treatment for Niemann-Pick disease appears safe
Niemann-Pick disease A or B in four pediatric patients and SMPD1 mutation carrier frequency in the Mexican population
NIH teams with industry to develop treatments for Niemann-Pick Type C disease.
Gene therapy shows promise for treating Niemann-Pick disease type C1
Zevra Therapeutics – NNPDF
IJMS, Free Full-Text
A Practical Chromogenic Procedure for the Detection of Homozygotes and Heterozygous Carriers of Niemann-Pick Disease
Newborn screening test developed for rare, deadly neurological disorder – Washington University School of Medicine in St. Louis
IJMS, Free Full-Text
Niemann-Pick Disease, A Pipeline Analysis Report 2018, Technavio
Juvenile CLN3 disease is a lysosomal cholesterol storage disorder: similarities with Niemann-Pick type C disease - eBioMedicine
Lithium ameliorates Niemann-Pick C1 disease phenotypes by impeding STING/SREBP2 activation - ScienceDirect
