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
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Frontiers Pre-clinical Mouse Models of Neurodegenerative Lysosomal Storage Diseases
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IJMS, Free Full-Text
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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
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Niemann-Pick disease type C: Video & Anatomy
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Valley boy battling rare disease getting stronger
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Niemann-Pick disease Information
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Juvenile CLN3 disease is a lysosomal cholesterol storage disorder: similarities with Niemann-Pick type C disease - eBioMedicine
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NPC1 enables cholesterol mobilization during long‐term potentiation that can be restored in Niemann–Pick disease type C by CYP46A1 activation
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Why are people fighting over a promising treatment for a fatal childhood disease?, Science
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Experimental Treatment for Niemann-Pick Disease Type C1 Appears Safe, Effective
mRNA Treatment Rescues Niemann–Pick Disease Type C1 in Patient Fibroblasts
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Acid Sphingomyelinase Deficiency disease: Malacards - Research Articles, Drugs, Genes, Clinical Trials
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Parents of twins with Niemann-Pick find a treatment
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Correlation of age of onset and clinical severity in Niemann–Pick disease type C1 with lysosomal abnormalities and gene expression
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