Duvoglustat HCl Increases Systemic and Tissue Exposure of Active Acid α-Glucosidase in Pompe Patients Co-administered with Alglucosidase α

Duvoglustat HCl (AT2220, 1-deoxynojirimycin) is an investigational pharmacological chaperone for the treatment of acid α-glucosidase (GAA) deficiency, which leads to the lysosomal storage disorder Pompe disease, which is characterized by progressive accumulation of lysosomal glycogen primarily in he...

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Detalles Bibliográficos
Autores principales: Kishnani, Priya, Tarnopolsky, Mark, Roberts, Mark, Sivakumar, Kumarswamy, Dasouki, Majed, Dimachkie, Mazen M., Finanger, Erika, Goker-Alpan, Ozlem, Guter, Karl A., Mozaffar, Tahseen, Pervaiz, Muhammad Ali, Laforet, Pascal, Levine, Todd, Adera, Matthews, Lazauskas, Richard, Sitaraman, Sheela, Khanna, Richie, Benjamin, Elfrida, Feng, Jessie, Flanagan, John J., Barth, Jay, Barlow, Carrolee, Lockhart, David J., Valenzano, Kenneth J., Boudes, Pol, Johnson, Franklin K., Byrne, Barry
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2017
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5417791/
https://www.ncbi.nlm.nih.gov/pubmed/28341561
http://dx.doi.org/10.1016/j.ymthe.2017.02.017

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5417791/
https://www.ncbi.nlm.nih.gov/pubmed/28341561
http://dx.doi.org/10.1016/j.ymthe.2017.02.017

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