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Steven A. Silber
Researcher at Johnson & Johnson
Publications - 3
Citations - 382
Steven A. Silber is an academic researcher from Johnson & Johnson. The author has contributed to research in topics: Expanded access & Disease. The author has an hindex of 3, co-authored 3 publications receiving 290 citations.
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Journal ArticleDOI
Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1–2 trial
Daniel S. Ory,Elizabeth A. Ottinger,Nicole Y. Farhat,Kelly A. King,Xuntian Jiang,Lisa Weissfeld,Elizabeth Berry-Kravis,Cristin Davidson,Simona Bianconi,Lee Ann Keener,Ravichandran Rao,Ariane Soldatos,Rohini Sidhu,Kimberly A Walters,Xin Xu,Audrey Thurm,Beth Solomon,William J. Pavan,Bernardus Nicolaas Machielse,Mark L. Kao,Steven A. Silber,John C. McKew,Carmen C. Brewer,Charles H. Vite,Steven U. Walkley,Christopher P. Austin,Forbes D. Porter +26 more
TL;DR: This open-label, dose-escalation phase 1-2a study gave monthly intrathecal HPβCD to participants with NPC1 with neurological manifestation at the National Institutes of Health and evaluated Serum and CSF 24(S)-hydroxycholesterol (24[S]-HC), which serves as a biomarker of target engagement, andCSF protein biomarkers were evaluated.
Journal ArticleDOI
Collaborative Development of 2-Hydroxypropyl-β-Cyclodextrin for the Treatment of Niemann-Pick Type C1 Disease
Elizabeth A. Ottinger,Mark L. Kao,Nuria Carrillo-Carrasco,Nicole M. Yanjanin,Roopa Kanakatti Shankar,Marjo Janssen,Marcus E. Brewster,Ilona Scott,Xin Xu,Jim Cradock,Pramod S. Terse,Seameen Dehdashti,Juan J. Marugan,Wei Zheng,Lili M Portilla,Alan Hubbs,William J. Pavan,John D. Heiss,Charles H. Vite,Steven U. Walkley,Daniel S. Ory,Steven A. Silber,Forbes D. Porter,Christopher P. Austin,John C. McKew +24 more
TL;DR: How this collaborative effort helped to overcome scientific, clinical and financial challenges facing the development of new drug treatments for rare and neglected diseases, and how it will incentivize the commercialization of HP-β-CD for the benefit of the NPC patient community are discussed are discussed.
Journal ArticleDOI
Flubendazole as a macrofilaricide: History and background.
TL;DR: Research has recently been devoted to the development of new formulations that would afford high oral bioavailability of FBZ, paving the way for potential clinical development of this repurposed drug for the treatment of human filariases.