ICV-administered BMN 250 (NAGLU-IGF2) is well tolerated and...

Molecular Genetics and Metabolism 2018 / 02 Vol. 123; Iss. 2

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)

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
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Volume:
123
Language:
english
Journal:
Molecular Genetics and Metabolism
DOI:
10.1016/j.ymgme.2017.12.270
Date:
February, 2018
File:
PDF, 55 KB
english, 2018
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