O
Ozlem Goker-Alpan
Researcher at National Institutes of Health
Publications - 139
Citations - 4253
Ozlem Goker-Alpan is an academic researcher from National Institutes of Health. The author has contributed to research in topics: Enzyme replacement therapy & Medicine. The author has an hindex of 28, co-authored 108 publications receiving 3528 citations.
Papers
More filters
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
Glucocerebrosidase mutations in subjects with parkinsonism
TL;DR: The findings suggest that mutations in glucocerebrosidase may be a risk factor for the development of parkinsonism.
Journal ArticleDOI
Parkinsonism among Gaucher disease carriers.
Ozlem Goker-Alpan,Raphael Schiffmann,Mary E. LaMarca,Robert L. Nussbaum,Aideen M. McInerney-Leo,Ellen Sidransky +5 more
TL;DR: Observations indicate that mutant glucocerebrosidase, even in heterozygotes, may be a risk factor for the development of parkinsonism, and will provide insights into the genetics, pathogenesis, and treatment of Parkinson disease.
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
Glucocerebrosidase mutations are an important risk factor for Lewy body disorders
Ozlem Goker-Alpan,Benoit I. Giasson,Michael J. Eblan,J. Nguyen,Howard I. Hurtig,Virginia M.-Y. Lee,John Q. Trojanowski,Ellen Sidransky +7 more
TL;DR: The glucocerebrosidase gene was examined in 75 autopsy specimens with different synucleinopathies and identified mutations in 23% of cases of dementia with Lewy bodies, expanding on previous findings in subjects with Parkinson disease.