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Collaborative research efforts drive therapeutic advancements for free sialic acid storage disorder (FSASD) Malicdan, May Christine; Adams, David R.; Brooks, P. J.; Dobrenis, Konstantin; Gahl, William A.; Gasnier, Bruno; Hackbarth, Mary E.; Hossain, Mahin; Leoyklang, Petcharat; Anne-Longin, Christine; Paavola, Liisa; Platt, Frances; Pollard, Laura; Reimer, Richard; Sabir, Marya S.; Wang, Raymond Y.; Walkley, Steven; Wasserstein, Melissa; Huizing, Marjan 分享 收藏
A concerted action to explore therapies for free sialic acid storage disease Huizing, Marjan; Walkley, Steven U.; Wasserstein, Melissa; Anne-Longin, Christine; Wang, Raymond Y.; Reimer, Richard; Pollard, Laura; Paavola, Liisa; Malicdan, May Christine; Sabir, Marya S.; Leoyklang, Petcharat; Adams, David R.; Gahl, William A. 分享 收藏
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2-Hydroxypropyl-β-cyclodextrin is the active component in a triple combination formulation for treatment of Niemann-Pick C1 disease Davidson, Jessica; Molitor, Elizabeth; Moores, Samantha; Gale, Sarah E.; Subramanian, Kanagaraj; Jiang, Xuntian; Sidhu, Rohini; Kell, Pamela; Zhang, Jesse; Fujiwara, Hideji; Davidson, Cristin; Helquist, Paul; Melancon, Bruce J.; Grigalunas, Michael; Liu, Gang; Salahi, Farbod; Wiest, Olaf; Xu, Xin; Porter, Forbes D.; Pipalia, Nina H.; Cruz, Dana L.; Holson, Edward B.; Schaffer, Jean E.; Walkley, Steven U.; Maxfield, Frederick R.; Ory, Daniel S. 分享 收藏
Intellectual and developmental disabilities research centers: Fifty years of scientific accomplishments Walkley, Steven U.; Abbeduto, Leonard; Batshaw, Mark L.; Bhattacharyya, Anita; Bookheimer, Susan Y.; Christian, Bradley T.; Constantino, John N.; de Vellis, Jean; Doherty, Daniel A.; Nelson, David L.; Piven, Joseph; Poduri, Annapurna; Pomeroy, Scott L.; Samaco, Rodney C.; Zoghbi, Huda Y.; Guralnick, Michael J. 分享 收藏
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Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1-2 trial Ory, Daniel S.; Ottinger, Elizabeth A.; Farhat, Nicole Yanjanin; King, Kelly A.; Jiang, Xuntian; Weissfeld, Lisa; Berry-Kravis, Elizabeth; Davidson, Cristin D.; Bianconi, Simona; Keener, Lee Ann; Rao, Ravichandran; Soldatos, Ariane; Sidhu, Rohini; Walters, Kimberly A.; Xu, Xin; Thurm, Audrey; Solomon, Beth; Pavan, William J.; Machielse, Bernardus N.; Kao, Mark; Silber, Steven A.; Mckew, John C.; Brewer, Carmen C.; Vite, Charles H.; Walkley, Steven U.; Austin, Christopher P.; Porter, Forbes D. 分享 收藏
N-butyldeoxynojirimycin delays motor deficits, cerebellar microgliosis, and Purkinje cell loss in a mouse model of mucolipidosis type IV Boudewyn, Lauren C.; Sikora, Jakub; Kuchar, Ladislav; Ledvinova, Jana; Grishchuk, Yulia; Wang, Shirley L.; Dobrenis, Kostantin; Walkley, Steven U. 分享 收藏
Cyclodextrin has conflicting actions on autophagy flux in vivo in brains of normal and Alzheimer model mice Yang, Dun-Sheng; Stavrides, Philip; Kumar, Asok; Jiang, Ying; Mohan, Panaiyur S.; Ohno, Masuo; Dobrenis, Kostantin; Davidson, Cristin D.; Saito, Mitsuo; Pawlik, Monika; Huo, Chunfeng; Walkley, Steven U.; Nixon, Ralph A. 分享 收藏
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Efficacy and ototoxicity of different cyclodextrins in Niemann-Pick C disease Davidson, Cristin D.; Fishman, Yonatan I.; Puskas, Istvan; Szeman, Julianna; Sohajda, Tamas; McCauliff, Leslie A.; Sikora, Jakub; Storch, Judith; Vanier, Marie T.; Szente, Lajos; Walkley, Steven U.; Dobrenis, Kostantin 分享 收藏
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Phase 1/2 evaluation of intrathecal 2-hydroxypropyl-β-cyclodextrin for the treatment of Niemann-Pick disease type C1 Porter, Forbes D.; Farhat, Nicole Y.; Ottinger, Elizabeth A.; McKew, John C.; Weissfeld, Lisa; Machielse, Ben; Berry-Kavis, Elizabeth M.; Vite, Charles H.; Walkley, Steven U.; Ory, Daniel S. 分享 收藏
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Different cyclodextrins for the treatment of Niemann-Pick disease type C Davidson, Cristin; Fishman, Yonatan; Puskas, Istvan; Szeman, Julliana; Sohajda, Tomas; Sikora, Jakub; Vanier, Marie T.; Szente, Lajos; Walkley, Steven U.; Dobrenis, Kostantin 分享 收藏
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Niemann-Pick C1 Is Essential for Ebolavirus Replication and Pathogenesis In Vivo Herbert, Andrew S.; Davidson, Cristin; Kuehne, Ana I.; Bakken, Russell; Braigen, Stephen Z.; Gunn, Kathryn E.; Whelan, Sean P.; Brummelkamp, Thijn R.; Twenhafel, Nancy A.; Chandran, Kartik; Walkley, Steven U.; Dye, John M. 分享 收藏
Intracisternal cyclodextrin prevents cerebellar dysfunction and Purkinje cell death in feline Niemann-Pick type C1 disease Vite, Charles H.; Bagel, Jessica H.; Swain, Gary P.; Prociuk, Maria; Sikora, Tracey U.; Stein, Veronika M.; O'Donnell, Patricia; Ruane, Therese; Ward, Sarah; Crooks, Alexandra; Li, Su; Mauldin, Elizabeth; Stellar, Susan; De Meulder, Marc; Kao, Mark L.; Ory, Daniel S.; Davidson, Cristin; Vanier, Marie T.; Walkley, Steven U. 分享 收藏