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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Publicado en:Mol Ther
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: Artigo
Lenguaje:Inglês
Publicado: American Society of Gene & Cell Therapy 2017
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Original Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC5417791/
https://ncbi.nlm.nih.gov/pubmed/28341561
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2017.02.017
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