METHODS AND COMPOSITIONS FOR TREATING HUNTER SYNDROME

The present invention provides, among other things, compositions and methods for CNS delivery of ldursulfase-beta, a human recombinant iduronate-2-sulfatase protein, for effective treatment of Hunter Syndrome. The compositions and methods provided by the present invention effectively reduce symptoms...

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Bibliographische Detailangaben
Hauptverfasser: JIN, THONG-GYU, LEE, BYOUNG-JU, BYUN, HAN-YEUL, OKUYAMA, TORAYUKI, SEO, JIN-WOOK, LEE, KYUHYUN, KIM, YONGUL, JANG, IN-YOUNG
Format: Patent
Sprache:eng
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Beschreibung
Zusammenfassung:The present invention provides, among other things, compositions and methods for CNS delivery of ldursulfase-beta, a human recombinant iduronate-2-sulfatase protein, for effective treatment of Hunter Syndrome. The compositions and methods provided by the present invention effectively reduce symptoms not only in brain and spinal cord but also in peripheral tissues including heart, liver, spleen, lung, and kidney.