MPS II Research Highlights: WORLDSymposium 2022
18 mar 2022 ·
21 min. 37 sec.
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Descrizione
This accredited CME activity, led by Barbara Burton, MD, Professor of Pediatrics at Northwestern University Feinberg School of Medicine highlights the latest research about Mucopolysaccharidosis type II (MPS II; Hunter...
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This accredited CME activity, led by Barbara Burton, MD, Professor of Pediatrics at Northwestern University Feinberg School of Medicine highlights the latest research about Mucopolysaccharidosis type II (MPS II; Hunter syndrome) presented at WORLDSymposium 2022 and provides expert analysis of its clinical relevance for busy members of the care team to help them care for patients they may encounter with this rare condition.
MPS II; Hunter syndrome is a rare, progressive lysosomal disease caused by deficient activity of iduronate-2-sulfatase, attributable to pathogenic variants of the iduronate-2-sulfatase gene (IDS). Course facial features and skeletal irregularities are the dominant symptoms of the periphery but of great concern is the central symptoms (cognitive decline, seizures) that occur in the more severe cases. Current therapies options include enzyme replace therapy but newer treatment options are in development, including treatments that may address the central symptoms. Supported by an educational grant from Takeda Pharmaceuticals U.S.A. Inc.
For complete activity information and to obtain CME credit, please go to https://checkrare.com/learning-center/courses/
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MPS II; Hunter syndrome is a rare, progressive lysosomal disease caused by deficient activity of iduronate-2-sulfatase, attributable to pathogenic variants of the iduronate-2-sulfatase gene (IDS). Course facial features and skeletal irregularities are the dominant symptoms of the periphery but of great concern is the central symptoms (cognitive decline, seizures) that occur in the more severe cases. Current therapies options include enzyme replace therapy but newer treatment options are in development, including treatments that may address the central symptoms. Supported by an educational grant from Takeda Pharmaceuticals U.S.A. Inc.
For complete activity information and to obtain CME credit, please go to https://checkrare.com/learning-center/courses/
Informazioni
| Autore | Peter Ciszewski, CheckRare |
| Organizzazione | CheckRare |
| Sito | - |
| Tag |
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