ICV-administered BMN 250 (NAGLU-IGF2) is well tolerated and reduces heparan sulfate accumulation in the CNS of subjects with Sanfilippo syndrome type B (MPS IIIB)

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Bibliographic Details
Published in:Molecular genetics and metabolism 2018-02, Vol.123 (2), p.S102-S102
Main Authors: Muschol, Nicole, Cleary, Maureen, Couce, Maria Luz, Shaywitz, Adam J., Cahan, Heather, Grover, Anita, Maricich, Stephen M., Melton, Andrew, Smith, Lynn, de Castro Lopez, Maria Jose
Format: Article
Language:English
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ISSN:1096-7192
1096-7206
DOI:10.1016/j.ymgme.2017.12.270