Experimental treatment for Niemann-Pick disease appears safe
Por um escritor misterioso
Last updated 21 março 2025

An experimental drug has appeared to slow the progression of Niemann-Pick disease the drug 2-hydroxypropyl-beta-cyclodextrin is being tested

Tralesinidase Alfa Enzyme Replacement Therapy Prevents Disease Manifestations in a Canine Model of Mucopolysaccharidosis Type IIIB

Health Tip on New Treatment that Shows Promise in Niemann-Pick Type C Disorder - Health Tips

A Practical Chromogenic Procedure for the Detection of Homozygotes and Heterozygous Carriers of Niemann-Pick Disease

Correlation of age of onset and clinical severity in Niemann–Pick disease type C1 with lysosomal abnormalities and gene expression

Psychiatric and Cognitive Symptoms Associated with Niemann-Pick Type C Disease: Neurobiology and Management

Adenosine A2A receptor stimulation restores cell functions and differentiation in Niemann-Pick type C-like oligodendrocytes

IJMS, Free Full-Text

Niemann–Pick type C disease: cellular pathology and pharmacotherapy - Wheeler - 2020 - Journal of Neurochemistry - Wiley Online Library

Acid Sphingomyelinase Deficiency disease: Malacards - Research Articles, Drugs, Genes, Clinical Trials

Niemann-Pick disease - News, Articles etc. - European Pharmaceutical Review

Niemann-Pick disease Information

Niemann-Pick Disease Type C (NP-C) Gene Therapy Activation Meeting (GAM) - International Niemann-Pick Disease Alliance (INPDA)
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