Gene therapy shows promise for treating Niemann-Pick disease type
Por um escritor misterioso
Descrição
NIH mouse study could lead to human clinical trials.
Niemann-Pick Disease Type C Gene Therapy Gets Rare Pediatric
Current gene editing strategies for the mucopolysaccharidoses
IJMS, Free Full-Text
Niemann-Pick disease type C: Video & Anatomy
Advancements in Niemann-Pick Disease Type C Treatments
Frontiers Pre-clinical Mouse Models of Neurodegenerative
Niemann–Pick type C disease as proof‐of‐concept for intelligent
Investigation of 2-Hydroxypropyl-β-Cyclodextrin Treatment in a
PDF) A therapy with miglustat, 2-hydroxypropyl-ß-cyclodextrin and
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por adulto (o preço varia de acordo com o tamanho do grupo)