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- Title
Highly phosphomannosylated enzyme replacement therapy for GM2 gangliosidosis.
- Authors
Tsuji, Daisuke; Akeboshi, Hiromi; Matsuoka, Kazuhiko; Yasuoka, Hiroko; Miyasaki, Eri; Kasahara, Yoshiko; Kawashima, Ikuo; Chiba, Yasunori; Jigami, Yoshifumi; Taki, Takao; Sakuraba, Hitoshi; Itoh, Kohji
- Abstract
The article examines the highly phosphomannosylated enzyme replacement therapy (ERT) for GM2 gangliosidoses. It states that the novel recombinant human lysosomal b-hexosaminidase A (HexA) has been developed for ERT for Tay-Sachs and Sandhoff diseases that is caused by HexA deficiency. Further it states that lysosome-directed recombinant human enzyme drug has a potential to improve the motor dysfunction and the lysosomal storage diseases (LSDs) patients with neurological manifestations.
- Subjects
CLINICAL medicine; NEUROLOGICAL disorders; ENZYMES; GANGLIOSIDOSES; THERAPEUTICS; NEUROLOGIC manifestations of general diseases
- Publication
Annals of Neurology, 2011, Vol 69, Issue 4, p691
- ISSN
0364-5134
- Publication type
Article
- DOI
10.1002/ana.22262