Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active alpha-Galactosidase A in Fabry Patients when Co-Administered with Infused Agalsidase

David G Warnock; Daniel G Bichet; Myrl Holida; Ozlem Goker-Alpan; Kathy Nicholls; Mark Thomas; Francois Eyskens; Suma Shankar; Mathews Adera; Sheela Sitaraman; Richie Khanna; John J Flanagan; Brandon A Wustman; Jay Barth; Carrolee Barlow; Kenneth J Valenzano; David J Lockhart; Pol Boudes; Franklin K Johnson

Journal article

Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active alpha-Galactosidase A in Fabry Patients when Co-Administered with Infused Agalsidase

David G Warnock, Daniel G Bichet, Myrl Holida, Ozlem Goker-Alpan, Kathy Nicholls, Mark Thomas, Francois Eyskens, Suma Shankar, Mathews Adera, Sheela Sitaraman, Richie Khanna, John J Flanagan, Brandon A Wustman, Jay Barth, Carrolee Barlow, Kenneth J Valenzano, David J Lockhart, Pol Boudes, Franklin K Johnson

PLOS ONE | PUBLIC LIBRARY SCIENCE | Published : 2015

DOI: 10.1371/journal.pone.0134341

University of Melbourne Researchers

Kathleen Nicholls Author Medicine - Royal Melbourne Hospital

Grants

Funding Acknowledgements

This clinical trial, AT1001-013, its design, execution, data analysis, report writing, and manuscript preparation was funded by Amicus Therapeutics and GSK. Several authors left Amicus subsequent to the preparation of this manuscript. JJF went to Arvinas Inc. BAW and DJL went to OrPhi Therapeutics. CB went to The Parkinson's Institute and Clinical Center. PB went to Cymabay Therapeutics. While these companies now provide support in the form of salaries for authors JJF, BAW, DJL, CB, and PB, they in no way provided any support for this clinical trial. Clinical Research Investigators OGA and MT are affiliated with private companies which, however, did not provide support for this clinical trial. The specific roles of these authors are articulated in the "author contributions" section.