1. Agalsidase alfa for the treatment of fabry disease: a closer look. (June 2014) Authors: Lipinski, Shawn E; Komlodi-Pasztor, Edina; Goker-Alpan, Ozlem Journal: Clinical investigation Issue: Volume 4:Number 6(2014) Page Start: 567 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
2. Clinical course and prognosis in patients with Gaucher disease and parkinsonism. (April 2016) Authors: Lopez, Grisel; Kim, Jenny; Wiggs, Edythe; Cintron, Dahima; Groden, Catherine; Tayebi, Nahid; Mistry, Pramod K.; Pastores, Gregory M.; Zimran, Ari; Goker-Alpan, Ozlem; Sidransky, Ellen Journal: Neurology Issue: Volume 2:Number 2(2016) Page Start: Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
3. Gaucher Disease and the Synucleinopathies. (28th May 2006) Authors: Hruska, Kathleen S.; Goker-Alpan, Ozlem; Sidransky, Ellen Journal: Journal of biomedicine and biotechnology Issue: Volume 2006(2006) Page Start: Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
4. Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease. (2nd August 2022) Authors: Dimachkie, Mazen M.; Barohn, Richard J.; Byrne, Barry; Goker-Alpan, Ozlem; Kishnani, Priya S.; Ladha, Shafeeq; Laforêt, Pascal; Mengel, Karl Eugen; Peña, Loren D.M.; Sacconi, Sabrina; Straub, Volker; Trivedi, Jaya; Van Damme, Philip; van der Ploeg, Ans T.; Vissing, John; Young, Peter; Haack, Kris... Journal: Neurology Issue: Volume 99:Number 5(2022) Page Start: e536 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
5. Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study. Issue 4 (10th November 2016) Authors: Hughes, Derralynn A; Nicholls, Kathleen; Shankar, Suma P; Sunder-Plassmann, Gere; Koeller, David; Nedd, Khan; Vockley, Gerard; Hamazaki, Takashi; Lachmann, Robin; Ohashi, Toya; Olivotto, Iacopo; Sakai, Norio; Deegan, Patrick; Dimmock, David; Eyskens, François; Germain, Dominique P; Goker-Alpan, O... Journal: Journal of medical genetics Issue: Volume 54:Issue 4(2017) Page Start: 288 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
6. P0062GLUCOSYLCERAMIDE SYNTHASE INHIBITION WITH VENGLUSTAT IN CLASSIC FABRY DISEASE PATIENTS LEADS TO PROGRESSIVE REDUCTION OF ENDOTHELIAL CELL GLOBOTRIAOSYLCERAMIDE INCLUSION VOLUME. (6th June 2020) Authors: Mauer, Michael; Goker-Alpan, Ozlem; Germain, Dominique P; Wilcox, William; Hopkin, Robert J; Lukina, Elena; Geberhiwot, Tarekegn; Deegan, Patrick; Tylki-Szymanska, Anna; Sensinger, Charlotte; Zaher, Atef; Hailman, Eric; Modur, Vijay; Najafian, Behzad Journal: Nephrology dialysis transplantation Issue: Volume 35(2020)Supplement 3 Page Start: Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
7. Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial. Issue 12 (December 2021) Authors: Diaz-Manera, Jordi; Kishnani, Priya S; Kushlaf, Hani; Ladha, Shafeeq; Mozaffar, Tahseen; Straub, Volker; Toscano, Antonio; van der Ploeg, Ans T; Berger, Kenneth I; Clemens, Paula R; Chien, Yin-Hsiu; Day, John W; Illarioshkin, Sergey; Roberts, Mark; Attarian, Shahram; Borges, Joao Lindolfo; Bouhou... Journal: Lancet neurology Issue: Volume 20:Issue 12(2021) Page Start: 1012 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
8. Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial. Issue 12 (December 2021) Authors: Schoser, Benedikt; Roberts, Mark; Byrne, Barry J; Sitaraman, Sheela; Jiang, Hai; Laforêt, Pascal; Toscano, Antonio; Castelli, Jeff; Díaz-Manera, Jordi; Goldman, Mitchell; van der Ploeg, Ans T; Bratkovic, Drago; Kuchipudi, Srilakshmi; Mozaffar, Tahseen; Kishnani, Priya S; Sebok, Agnes; Pestronk, A... Journal: Lancet neurology Issue: Volume 20:Issue 12(2021) Page Start: 1027 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
9. Tetrahydrobiopterin deficiency in the pathogenesis of Fabry disease. (2nd February 2017) Authors: Shen, Jin-Song; Arning, Erland; West, Michael L.; Day, Taniqua S.; Chen, Shuyuan; Meng, Xing-Li; Forni, Sabrina; McNeill, Nathan; Goker-Alpan, Ozlem; Wang, Xuan; Ashcraft, Paula; Moore, David F.; Cheng, Seng H.; Schiffmann, Raphael; Bottiglieri, Teodoro Journal: Human molecular genetics Issue: Volume 26:Number 6(2017:Mar. 15) Page Start: 1182 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗