Niemann-Pick Disease (NPD) is a heterogeneous group of autosomal recessive disorders characterized by progressive accumulation of sphingomyelin and cholesterol in lysosomes. Six types of NPD have been described based on clinical presentation and involved organs. The primary defect in NPD types A and
Five young patients with Niemann-Pick disease type B were treated with repeated implantations of amniotic epithelial cells, as a source of exogenous sphingomyelinase. This treatment abolished the recurrent infections, mainly of the respiratory tract, and led to other improvements of the general
OBJECTIVE
Enzyme replacement therapy with olipudase alfa (recombinant human acid sphingomyelinase) is being developed for Niemann-Pick disease type B (NPD B).
METHODS
A single-center, open-label, nonrandomized, single-ascending-dose trial evaluated the safety of intravenous olipudase alfa (0.03-1.0
The most complete medicinal herbs database backed by science
Works in 55 languages
Herbal cures backed by science
Herbs recognition by image
Interactive GPS map - tag herbs on location (coming soon)
Read scientific publications related to your search
Search medicinal herbs by their effects
Organize your interests and stay up do date with the news research, clinical trials and patents
Type a symptom or a disease and read about herbs that might help, type a herb and see diseases and symptoms it is used against. *All information is based on published scientific research