METHODS AND COMPOSITIONS FOR CNS DELIVERY OF HEPARAN N-SULFATASE

The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention includes a stable formulation for direct CNS intrathecal administration comprising a he...

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Main Authors SHAHROKH, ZAHRA, VERNAGLIA, BRIAN, PAN, JING, PFEIFER, RICHARD, TSUNG, JAMIE, ZHU, GAOZHONG, TEREW, JENNIFER, CALIAS, PERICLES, JIANG, YUAN, NATOLI, FARAH
Format Patent
LanguageEnglish
French
German
Published 09.04.2014
Subjects
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
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Summary:The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention includes a stable formulation for direct CNS intrathecal administration comprising a heparan N-sulfatase (HNS) protein, salt, and a polysorbate surfactant for the treatment of Sanfilippo Syndrome Type A.
Bibliography:Application Number: EP20110799034