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

Molecular Therapy Methods and Clinical Development

Volumn 6, Issue , 2017, Pages 43-53

  Aoyagi Scharber, Mika ^a   Crippen Harmon, Danielle ^a   Lawrence, Roger ^a   Vincelette, Jon ^a   Yogalingam, Gouri ^a   Prill, Heather ^a   Yip, Bryan K ^a   Baridon, Brian ^a   Vitelli, Catherine ^a   Lee, Amanda ^a   Gorostiza, Olivia ^a   Adintori, Evan G ^a   Minto, Wesley C ^a   Van Vleet, Jeremy L ^a   Yates, Bridget ^a   Rigney, Sara ^a   Christianson, Terri M ^a   Tiger, Pascale M N ^a   Lo, Melanie J ^a   Holtzinger, John ^a   Fitzpatrick, Paul A ^a   LeBowitz, Jonathan H ^a   Bullens, Sherry ^a   Crawford, Brett E ^a   Bunting, Stuart ^a   more..

^a BIOMARIN PHARMACEUTICAL INC   (United States)

Author keywords

enzyme replacement therapy; glycosaminoglycan; intracerebroventricular delivery; lysosomal storage disorder; mental retardation; MPS IIIB; mucopolysaccharidosis IIIB; neurodegeneration; Sanfilippo B

Indexed keywords

ALPHA N ACETYLGLUCOSAMINIDASE; BMN 250; HEPARAN SULFATE; SOMATOMEDIN B;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; ASTROCYTOSIS; BRAIN REGION; CELLULAR DISTRIBUTION; CONTROLLED STUDY; DOSE RESPONSE; DRUG ACCUMULATION; DRUG DISTRIBUTION; DRUG UPTAKE; LYSOSOME STORAGE DISEASE; MOUSE; NERVOUS SYSTEM INFLAMMATION; NEUROPATHOLOGY; NONHUMAN; PRIORITY JOURNAL; SANFILIPPO SYNDROME;

EID: 85029310056     PISSN: None     EISSN: 23290501     Source Type: Journal    
DOI: 10.1016/j.omtm.2017.05.009     Document Type: Article

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