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)
| Autor: | 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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| Zdroj: | In Molecular Genetics and Metabolism February 2018 123(2):S102-S102 |
| Databáze: | ScienceDirect |
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