K
Khan Nedd
Publications - 9
Citations - 712
Khan Nedd is an academic researcher. The author has contributed to research in topics: Enzyme replacement therapy & Fabry disease. The author has an hindex of 6, co-authored 7 publications receiving 515 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
Safety and efficacy of alternative alglucosidase alfa regimens in Pompe disease
Laura E. Case,Carl Bjartmar,Claire Morgan,Robin Casey,Joel Charrow,John P. Clancy,Majed Dasouki,Stephanie DeArmey,Khan Nedd,Mary Nevins,Heidi Peters,Dawn Phillips,Zachary Spigelman,Cynthia J. Tifft,Priya S. Kishnani +14 more
TL;DR: Results were inconclusive but suggest that increased ERT dose may be beneficial in some patients with Pompe disease experiencing motor decline, and controlled studies are needed to clarify the benefits and risks of this strategy.
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.
Book ChapterDOI
Reduction of Plasma Globotriaosylsphingosine Levels After Switching from Agalsidase Alfa to Agalsidase Beta as Enzyme Replacement Therapy for Fabry Disease.
Ozlem Goker-Alpan,Michael J. Gambello,Gustavo Maegawa,Khan Nedd,Daniel Gruskin,Larry Blankstein,Neal J. Weinreb +6 more
TL;DR: Plasma lyso-GL-3 and GL-3 levels reduced after switching from agalidase alfa to agalsidase beta, indicating that agalsidsase beta has a greater pharmacodynamic effect on these markers at the recommended dose.