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Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active α-Galactosidase A in Fabry Patients when Co-Administered with Infused Agalsidase.
Warnock, David G; Bichet, Daniel G; Holida, Myrl; Goker-Alpan, Ozlem; Nicholls, Kathy; Thomas, Mark; Eyskens, Francois; Shankar, Suma; Adera, Mathews; Sitaraman, Sheela; Khanna, Richie; Flanagan, John J; Wustman, Brandon A; Barth, Jay; Barlow, Carrolee; Valenzano, Kenneth J; Lockhart, David J; Boudes, Pol; Johnson, Franklin K.
Affiliation
  • Warnock DG; University of Alabama at Birmingham School of Medicine Department of Medicine, Division of Nephrology, Birmingham, Alabama, United States of America.
  • Bichet DG; University of Montreal, Quebec, Canada.
  • Holida M; University of Iowa Children's Hospital, Iowa City, Iowa, United States of America.
  • Goker-Alpan O; LSD Research and Treatment Unit, O&O Alpan LLC, Fairfax, Virginia, United States of America.
  • Nicholls K; Department of Nephrology, Royal Melbourne Hospital, and University of Melbourne, Parkville, Victoria, Australia.
  • Thomas M; Linear Clinical Research Ltd, Queen Elizabeth II Medical Centre, and University of Western Australia, Nedlands, Western Australia.
  • Eyskens F; Universitair Ziekenhuis Antwerpen, Antwerpen, Belgium.
  • Shankar S; Emory University School of Medicine, Emory Genetics Clinic, Athens, Georgia, United States of America.
  • Adera M; Amicus Therapeutics, Cranbury, New Jersey, United States of America.
  • Sitaraman S; Amicus Therapeutics, Cranbury, New Jersey, United States of America.
  • Khanna R; Amicus Therapeutics, Cranbury, New Jersey, United States of America.
  • Flanagan JJ; Arvinas Inc, New Haven, Connecticut, United States of America.
  • Wustman BA; Institute and Clinical Center, Sunnyvale, California, United States of America.
  • Barth J; Amicus Therapeutics, Cranbury, New Jersey, United States of America.
  • Barlow C; Amicus Therapeutics, Cranbury, New Jersey, United States of America.
  • Valenzano KJ; Amicus Therapeutics, Cranbury, New Jersey, United States of America.
  • Lockhart DJ; Institute and Clinical Center, Sunnyvale, California, United States of America.
  • Boudes P; Cymabay Therapeutics, Newark, California, United States of America.
  • Johnson FK; Amicus Therapeutics, Cranbury, New Jersey, United States of America.
PLoS One ; 10(8): e0134341, 2015.
Article in En | MEDLINE | ID: mdl-26252393
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Full text: 1 Database: MEDLINE Main subject: Fabry Disease / 1-Deoxynojirimycin / Alpha-Galactosidase / Isoenzymes Type of study: Prognostic_studies Limits: Adult / Humans / Male / Middle aged Language: En Year: 2015 Type: Article
Fulltext
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PubMed Links
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Full text: 1 Database: MEDLINE Main subject: Fabry Disease / 1-Deoxynojirimycin / Alpha-Galactosidase / Isoenzymes Type of study: Prognostic_studies Limits: Adult / Humans / Male / Middle aged Language: En Year: 2015 Type: Article