Literature DB >> 27665929

Fabry disease: A pharmacological chaperone on the horizon.

Martina Gaggl1, Gere Sunder-Plassmann1.   

Abstract

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Year:  2016        PMID： 27665929     DOI： 10.1038/nrneph.2016.138

Source DB:  PubMed          Journal:  Nat Rev Nephrol        ISSN： 1759-5061            Impact factor:   28.314


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  9 in total

1.  Enzyme replacement therapy in Fabry disease: comparison of agalsidase alfa and agalsidase beta.

Authors:  Atul Mehta; Michael Beck; Christoph Kampmann; Andrea Frustaci; Dominique P Germain; Gregory M Pastores; Gere Sunder-Plassmann
Journal:  Mol Genet Metab       Date:  2008-08-12       Impact factor: 4.797

2.  Serum-Mediated Inhibition of Enzyme Replacement Therapy in Fabry Disease.

Authors:  Malte Lenders; Jörg Stypmann; Thomas Duning; Boris Schmitz; Stefan-Martin Brand; Eva Brand
Journal:  J Am Soc Nephrol       Date:  2015-04-30       Impact factor: 10.121

3.  Fabry disease: is there a role for enzyme replacement therapy?

Authors:  A B Mehta
Journal:  J Intern Med       Date:  2013-05-30       Impact factor: 8.989

4.  Migalastat: First Global Approval.

Authors:  Anthony Markham
Journal:  Drugs       Date:  2016-07       Impact factor: 9.546

Review 5.  Narrative review: Fabry disease.

Authors:  Joe T R Clarke
Journal:  Ann Intern Med       Date:  2007-03-20       Impact factor: 25.391

6.  Treatment of Fabry's Disease with the Pharmacologic Chaperone Migalastat.

Authors:  Dominique P Germain; Derralynn A Hughes; Kathleen Nicholls; Daniel G Bichet; Roberto Giugliani; William R Wilcox; Claudio Feliciani; Suma P Shankar; Fatih Ezgu; Hernan Amartino; Drago Bratkovic; Ulla Feldt-Rasmussen; Khan Nedd; Usama Sharaf El Din; Charles M Lourenco; Maryam Banikazemi; Joel Charrow; Majed Dasouki; David Finegold; Pilar Giraldo; Ozlem Goker-Alpan; Nicola Longo; C Ronald Scott; Roser Torra; Ahmad Tuffaha; Ana Jovanovic; Stephen Waldek; Seymour Packman; Elizabeth Ludington; Christopher Viereck; John Kirk; Julie Yu; Elfrida R Benjamin; Franklin Johnson; David J Lockhart; Nina Skuban; Jeff Castelli; Jay Barth; Carrolee Barlow; Raphael Schiffmann
Journal:  N Engl J Med       Date:  2016-08-11       Impact factor: 91.245

7.  Glycomimetic-based pharmacological chaperones for lysosomal storage disorders: lessons from Gaucher, GM1-gangliosidosis and Fabry diseases.

Authors:  Elena M Sánchez-Fernández; José M García Fernández; Carmen Ortiz Mellet
Journal:  Chem Commun (Camb)       Date:  2016-04-25       Impact factor: 6.222

8.  Safety and pharmacodynamic effects of a pharmacological chaperone on α-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinical studies.

Authors:  Dominique P Germain; Roberto Giugliani; Derralynn A Hughes; Atul Mehta; Kathy Nicholls; Laura Barisoni; Charles J Jennette; Alexander Bragat; Jeff Castelli; Sheela Sitaraman; David J Lockhart; Pol F Boudes
Journal:  Orphanet J Rare Dis       Date:  2012-11-24       Impact factor: 4.123

9.  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.

Authors:  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:  PLoS One       Date:  2015-08-07       Impact factor: 3.240
  9 in total
  2 in total

1.  Safety of switching to Migalastat from enzyme replacement therapy in Fabry disease: Experience from the Phase 3 ATTRACT study.

Authors:  Derralynn A Hughes; Kathleen Nicholls; Gere Sunder-Plassmann; Ana Jovanovic; Ulla Feldt-Rasmussen; Raphael Schiffmann; Robert Giugliani; Vipul Jain; Chris Viereck; Jeffrey P Castelli; Nina Skuban; Jay A Barth; Daniel G Bichet
Journal:  Am J Med Genet A       Date:  2019-03-28       Impact factor: 2.802

Review 2.  Carbohydrate-based drugs launched during 2000-2021.

Authors:  Xin Cao; Xiaojing Du; Heng Jiao; Quanlin An; Ruoxue Chen; Pengfei Fang; Jing Wang; Biao Yu
Journal:  Acta Pharm Sin B       Date:  2022-05-23       Impact factor: 14.903
  2 in total

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