Glucocerebrosidase mutations are an important risk factor for lewy body disorders von GOKER-ALPAN, O, GIASSON, B. I, EBLAN, M. J, NGUYEN, J, HURTIG, H. I, LEE, V. M.-Y, TROJANOWSKI, J. Q, SIDRANSKY, E

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Autoantibodies, T, B and Dendritic cell abnormalities in Postural Orthostatic Tachycardia Syndrome (POTS) von Abdallah, H, Vo, T, Alpan, O

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The Role of Dendritic Cells, B Cells, and M Cells in Gut-Oriented Immune Responses von Alpan, Oral, Rudomen, Gregory, Matzinger, Polly

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Phenotypic continuum in neuronopathic gaucher disease: an intermediate phenotype between type 2 and type 3 von Goker-Alpan, Ozlem, Schiffmann, Raphael, Park, Joseph K, Stubblefield, Barbara K, Tayebi, Nahid, Sidransky, Ellen

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Presenting signs and patient co-variables in Gaucher disease: outcome of the Gaucher Earlier Diagnosis Concensus (GED-C) Delphi initiative (vol 49, pg 578, 2019) von Mehta, A, Kuter, DJ, Salek, SS, Belmatoug, N, Bembi, B, Bright, J, Dahl, SV, Deodato, F, Di, Rocco M, Göker-Alpan, O

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Divergent phenotypes in Gaucher disease implicate the role of modifiers von Goker-Alpan, O, Hruska, K S, Orvisky, E, Kishnani, P S, Stubblefield, B K, Schiffmann, R, Sidransky, E

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Treatment of Fabry’s Disease with the Pharmacologic Chaperone Migalastat von Germain, Dominique P, Hughes, Derralynn A, Nicholls, Kathleen, Bichet, Daniel G, Giugliani, Roberto, Wilcox, William R, Feliciani, Claudio, Shankar, Suma P, Ezgu, Fatih, Amartino, Hernan, Bratkovic, Drago, Feldt-Rasmussen, Ulla, Nedd, Khan, Sharaf El Din, Usama, Lourenco, Charles M, Banikazemi, Maryam, Charrow, Joel, Dasouki, Majed, Finegold, David, Giraldo, Pilar, Goker-Alpan, Ozlem, Longo, Nicola, Scott, C. Ronald, Torra, Roser, Tuffaha, Ahmad, Jovanovic, Ana, Waldek, Stephen, Packman, Seymour, Ludington, Elizabeth, Viereck, Christopher, Kirk, John, Yu, Julie, Benjamin, Elfrida R, Johnson, Franklin, Lockhart, David J, Skuban, Nina, Castelli, Jeff, Barth, Jay, Barlow, Carrolee, Schiffmann, Raphael

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Parkinsonism among Gaucher disease carriers von Goker-Alpan, O, Schiffmann, R, LaMarca, M E, Nussbaum, R L, McInerney-Leo, A, Sidransky, E

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T.P.24 von Thurberg, B.L, van der Ploeg, A, Kissel, J.T, Schoser, B, Pestronk, A, Barohn, R.J, Goker-Alpan, O, Mozaffar, T, Pena, L.D.M, Simmons, Z, Straub, V, Young, P, Bjartmar, C

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CLINICAL ASSESSMENT OF SAFETY, PHARMACOKINETICS, AND EFFICACY OF IMMUNE GLOBULIN INTRAVENOUS 10% IN PEDIATRIC PATIENTS von Melamed, I., Walter, J., Alpan, O., Jhaveri, D., Koterba, A., Leiding, J.

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Presenting signs and patient co‐variables in Gaucher disease: outcome of the Gaucher Earlier Diagnosis Consensus (GED‐C) Delphi initiative von Mehta, Atul, Kuter, David J., Salek, Sam S., Belmatoug, Nadia, Bembi, Bruno, Bright, Jeremy, vom Dahl, Stephan, Deodato, Federica, Di Rocco, Maja, Göker‐Alpan, Ozlem, Hughes, Derralynn A., Lukina, Elena A., Machaczka, Maciej, Mengel, Eugen, Nagral, Aabha, Nakamura, Kimitoshi, Narita, Aya, Oliveri, Beatriz, Pastores, Gregory, Pérez‐López, Jordi, Ramaswami, Uma, Schwartz, Ida V., Szer, Jeff, Weinreb, Neal J., Zimran, Ari

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D020 ROLE OF CELLULAR CASPASES AND THERAPEUTIC POTENTIAL OF A PAN-CASPASE INHIBITOR, EMRICASAN, IN COVID-19 von Alpan, O., Gupta, R., Latterich, M., Hubka, M., Bukhari, Z., Ndhlovu, L.

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GBA1 -Associated Parkinson's Disease Is a Distinct Entity von Skrahin, Aliaksandr, Horowitz, Mia, Istaiti, Majdolen, Skrahina, Volha, Lukas, Jan, Yahalom, Gilad, Cohen, Mikhal E, Revel-Vilk, Shoshana, Goker-Alpan, Ozlem, Becker-Cohen, Michal, Hassin-Baer, Sharon, Svenningsson, Per, Rolfs, Arndt, Zimran, Ari

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Skin abnormalities as an early predictor of neurologic outcome in Gaucher disease von Holleran, WM, Ziegler, SG, Goker-Alpan, O, Eblan, MJ, Elias, PM, Schiffmann, R, Sidransky, E

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UNIPARENTAL DISOMY OF CHROMOSOME 1 CAUSING CONCURRENT CHARCOT-MARIE-TOOTH AND GAUCHER DISEASE TYPE 3 von BENKO, W. S, HRUSKA, K. S, NAGAN, N, GOKER-ALPAN, O, HART, P. S, SCHIFFMANN, R, SIDRANSKY, E

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T.P.46 A novel phase 2a study design to investigate drug-drug interactions between escalating doses of AT2220 (duvoglustat hydrochloride) and acid alpha-glucosidase in subjects wit... von Kishnani, P, Tarnopolsky, M, Sivakumar, K, Byrne, B, Goker-Alpan, O, Guter, K, Pervaiz, M, Dasouki, M, Levine, T, Roberts, M, Johnson, F.K, Valenzano, K.J, Lockhart, D.J, Boudes, P

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P372 Safety of home administration of cipaglucosidase alfa + miglustat in late-onset Pompe disease: results from multiple clinical trials von Byrne, B., Diaz-Manera, J., Goker-Alpan, O., Mozaffar, T., Wasfi, Y., Das, S Sitaraman, Fox, B., Holdbrook, F., Jain, V.

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The association between mutations in the lysosomal protein glucocerebrosidase and parkinsonism von DePaolo, John, Goker-Alpan, Ozlem, Samaddar, Ted, Lopez, Grisel, Sidransky, Ellen

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T.P.45 An ongoing phase 2a study to investigate drug–drug interactions between escalating doses of AT2220 (duvoglustat hydrochloride) and acid alpha glucosidase in subjects with Po... von Kishnani, P, Tarnopolsky, M, Sivakumar, K, Byrne, B, Goker-Alpan, O, Guter, K, Pervaiz, M, Dasouki, M, Levine, T, Roberts, M, Johnson, F.K, Sitaraman, S, Khanna, R, Flanagan, J, Sjoberg, E.R, Valenzano, K.J, Lockhart, D.J, Boudes, P

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Clinical Outcomes With Recombinant Human C1 Inhibitor In The Repeat Treatment Of Acute Attacks Of Hereditary Angioedema In North-american Patients von Zuraw, B, Craig, T, Lockey, R.F, Park, M, Grant, J, Suez, D, Schwartz, L.B, Riedl, M, Levy, R.J, Li, H.H, Edara, L, Bernstein, J.A, Moy, J, Offenberger, J, Szema, A, Engler, A, Rehman, S.M, Marbury, T.C, Alpan, O, Bahna, S.L, Davis, A, Sussman, G, Yang, W.H, Ritchie, B, Relan, A

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