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Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice

Molecular therapy. Methods & clinical development, 2017-09, Vol.6 (C), p.43-53

2017

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Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB

Proceedings of the National Academy of Sciences - PNAS, 2014-10, Vol.111 (41), p.14870-14875

2014

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Intracerebroventricular dosing of N-sulfoglucosamine sulfohydrolase in mucopolysaccharidosis IIIA mice reduces markers of brain lysosomal dysfunction

The Journal of biological chemistry, 2022-12, Vol.298 (12), p.102625-102625, Article 102625

2022

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Glycosylation independent lysosomal targeting of alpha-n-acetylglucosaminidase confers highly efficient enzyme uptake into critical cellular targets of disease pathogenesis in mucopolysaccharidosis type IIIB

Molecular genetics and metabolism, 2016-02, Vol.117 (2), p.S122-S122

2016

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Engineering of a recombinant NAGLU fusion protein with insulin-like growth factor 2 leads to improved cellular uptake via a glycosylation-independent lysosomal targeting pathway

Molecular genetics and metabolism, 2014-02, Vol.111 (2), p.S20-S20

2014

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New building blocks for the assembly of sequence selective molecular zippers

Chemical communications (Cambridge, England), 2003-01 (14), p.1642-1643

2003

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Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice

Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB

Intracerebroventricular dosing of N-sulfoglucosamine sulfohydrolase in mucopolysaccharidosis IIIA mice reduces markers of brain lysosomal dysfunction

Translational studies of intravenous and intracerebroventricular routes of administration for CNS cellular biodistribution for BMN 250, an enzyme replacement therapy for the treatment of Sanfilippo type B

Glycosylation independent lysosomal targeting of alpha-n-acetylglucosaminidase confers highly efficient enzyme uptake into critical cellular targets of disease pathogenesis in mucopolysaccharidosis type IIIB

Time- and dose-dependent normalization of pathological lysosomal storage and biochemistry in the mucopolysaccharidosis ΙΙΙΒ (MPS ΙΙΙΒ, Sanfilippo syndrome type Β) mouse model by intracerebroventricular enzyme replacement therapy with ΒΜΝ 250, a ΝAGLU-ΙGF2 fusion pro

Histologic characterization of the progression of central nervous system pathology in the mucopolysaccharidosis IIIB (MPS IIIB, Sanfilippo syndrome type B) mouse model and bio-distribution and efficacy of the intracerebroventricular enzyme replacement therapy, BMN 250

Engineering of a recombinant NAGLU fusion protein with insulin-like growth factor 2 leads to improved cellular uptake via a glycosylation-independent lysosomal targeting pathway

New building blocks for the assembly of sequence selective molecular zippers

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