Experimental treatment for Niemann-Pick disease appears safe

Por um escritor misterioso

Last updated 04 abril 2025

Experimental treatment for Niemann-Pick disease appears safe

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

Frontiers Pre-clinical Mouse Models of Neurodegenerative Lysosomal Storage Diseases

Experimental treatment for Niemann-Pick disease type C1 appears safe, effective

Push to spur more drugs for deadly rare diseases

Dermatologic Manifestations of Niemann-Pick Disease: Background, Pathophysiology, Etiology

mRNA Treatment Rescues Niemann–Pick Disease Type C1 in Patient Fibroblasts

NIH teams with industry to develop treatments for Niemann-Pick Type C disease.

Families plead with FDA for drug to treat Niemann-Pick Disease Type C

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

Frontiers Potential Disease-Modifying Effects of Lithium Carbonate in Niemann-Pick Disease, Type C1

Emerging Startups Take Aim at Lysosomal Storage Disorders

Enhanced Delivery and Effects of Acid Sphingomyelinase by ICAM-1-Targeted Nanocarriers in Type B Niemann-Pick Disease Mice - ScienceDirect

Niemann-Pick disease A or B in four pediatric patients and SMPD1 mutation carrier frequency in the Mexican population

From Life-Changing Clinical Trials to Running for Rare: Justin's Journey as a Rare Disease Father - National Organization for Rare Disorders

Gene therapy to the blood–brain barrier with resulting protein secretion as a strategy for treatment of Niemann Picks type C2 disease - Hede - 2021 - Journal of Neurochemistry - Wiley Online Library

Therapeutic intervention for Niemann–Pick type C disease must target

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