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Artigo Revisado por pares
BIBTEX RIS

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)

2018; Elsevier BV; Volume: 123; Issue: 2 Linguagem: Inglês

10.1016/j.ymgme.2017.12.270

ISSN

1096-7206

Autores

Nicole Muschol, Maureen Cleary, María L. Couce, Adam J. Shaywitz, Heather Cahan, Anita Grover, Stephen M. Maricich, Andrew C. Melton, Lynn Smith, María José de Castro López,

Tópico(s)

Hereditary Neurological Disorders

Referência(s)