J
Julie Yu
Researcher at Amicus Therapeutics
Publications - 13
Citations - 790
Julie Yu is an academic researcher from Amicus Therapeutics. The author has contributed to research in topics: Migalastat & Fabry disease. The author has an hindex of 5, co-authored 13 publications receiving 573 citations.
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Journal ArticleDOI
Treatment of Fabry’s Disease with the Pharmacologic Chaperone Migalastat
Dominique P. Germain,Derralynn Hughes,Kathleen Nicholls,Daniel G. Bichet,Roberto Giugliani,William R. Wilcox,Claudio Feliciani,Suma P. Shankar,Fatih Süheyl Ezgü,Hernan Amartino,Drago Bratkovic,Ulla Feldt-Rasmussen,Khan Nedd,Usama A Sharaf El Din,Charles Marques Lourenço,Maryam Banikazemi,Joel Charrow,Majed Dasouki,David N. Finegold,P 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 K. Johnson,David J. Lockhart,Nina Skuban,Jeff Castelli,Jay A. Barth,Carrolee Barlow,Raphael Schiffmann,Raphael Schiffmann +40 more
TL;DR: Among all randomly assigned patients with Fabry's disease (with mutant α-galactosidase forms that were suitable or not suitable for migalastat therapy), the percentage of patients who had a response at 6 months did not differ significantly between the migAlastat group and the placebo group.
Journal ArticleDOI
Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study
Derralynn Hughes,Kathleen Nicholls,Suma P. Shankar,Gere Sunder-Plassmann,David M. Koeller,Khan Nedd,Gerard Vockley,Takashi Hamazaki,Robin H. Lachmann,Toya Ohashi,Iacopo Olivotto,Norio Sakai,Patrick Deegan,David Dimmock,François Eyskens,Dominique P. Germain,Ozlem Goker-Alpan,Eric Hachulla,Ana Jovanovic,Charles Marques Lourenço,Ichiei Narita,Mark Thomas,William R. Wilcox,Daniel G. Bichet,Raphael Schiffmann,Elizabeth Ludington,Christopher Viereck,John Kirk,Julie Yu,Franklin K. Johnson,Pol Boudes,Elfrida R. Benjamin,David J. Lockhart,Carrolee Barlow,Nina Skuban,Jeffrey P. Castelli,Jay A. Barth,Ulla Feldt-Rasmussen +37 more
TL;DR: Migalastat offers promise as a first-in-class oral monotherapy alternative treatment to intravenous ERT for patients with Fabry disease and amenable mutations.
Journal ArticleDOI
The validation of pharmacogenetics for the identification of Fabry patients to be treated with migalastat.
Elfrida R. Benjamin,Maria Cecilia Della Valle,Xiaoyang Wu,Evan Katz,Farhana Pruthi,Sarah Bond,Benjamin Bronfin,Hadis Williams,Julie Yu,Daniel G. Bichet,Dominique P. Germain,Roberto Giugliani,Derralynn Hughes,Raphael Schiffmann,William R. Wilcox,Robert J. Desnick,John Kirk,Jay A. Barth,Carrolee Barlow,Kenneth J. Valenzano,Jeff Castelli,David J. Lockhart +21 more
TL;DR: The GLP HEK assay is a clinically validated method of identifying male and female Fabry patients for treatment with migalastat, a pharmacological chaperone that binds to specific mutant forms of α-galactosidase A to restore lysosomal activity.
Journal ArticleDOI
Long-term efficacy and safety of migalastat treatment in Fabry disease: 30-month results from the open-label extension of the randomized, phase 3 ATTRACT study.
Ulla Feldt-Rasmussen,Derralynn Hughes,Gere Sunder-Plassmann,Suma P. Shankar,Khan Nedd,Iacopo Olivotto,Damara Ortiz,Toya Ohashi,Takashi Hamazaki,Nina Skuban,Julie Yu,Jay A. Barth,Kathleen Nicholls +12 more
TL;DR: In patients with Fabry disease and amenable GLA variants, migalastat 150 mg QOD was well tolerated and demonstrated durable, long-term stability of renal function and reduction in LVMi.
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