Translational studies of intravenous and intracerebroventricular routes of administration for CNS cellular biodistribution for BMN 250, an enzyme replacement therapy for the treatment of Sanfilippo type B

BMN 250 is being developed as enzyme replacement therapy for Sanfilippo type B, a primarily neurological rare disease, in which patients have deficient lysosomal alpha-N-acetylglucosaminidase (NAGLU) enzyme activity. BMN 250 is taken up in target cells by the cation-independent mannose 6-phosphate r...

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Detalles Bibliográficos
Autores principales: Grover, Anita, Crippen-Harmon, Danielle, Nave, Lacey, Vincelette, Jon, Wait, Jill C. M., Melton, Andrew C., Lawrence, Roger, Brown, Jillian R., Webster, Katherine A., Yip, Bryan K., Baridon, Brian, Vitelli, Catherine, Rigney, Sara, Christianson, Terri M., Tiger, Pascale M. N., Lo, Melanie J., Holtzinger, John, Shaywitz, Adam J., Crawford, Brett E., Fitzpatrick, Paul A., LeBowitz, Jonathan H., Bullens, Sherry, Aoyagi-Scharber, Mika, Bunting, Stuart, O’Neill, Charles A., Pinkstaff, Jason, Bagri, Anil
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer US 2020
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7066106/
https://www.ncbi.nlm.nih.gov/pubmed/31942701
http://dx.doi.org/10.1007/s13346-019-00683-6

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