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The Platform Vector Gene Therapies Project: Increasing the Efficiency of Adeno-Associated Virus Gene Therapy Clinical Trial Startup.
Brooks PJ, Ottinger EA, Portero D, Lomash RM, Alimardanov A, Terse P, Xu X, Chandler RJ, Geist Hauserman J, Esposito E, Bönnemann CG, Venditti CP, Austin CP, Pariser A, Lo DC. Brooks PJ, et al. Among authors: pariser a. Hum Gene Ther. 2020 Oct;31(19-20):1034-1042. doi: 10.1089/hum.2020.259. Hum Gene Ther. 2020. PMID: 32993373 Free PMC article. No abstract available.
Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National Institutes of Health Platform Vector Gene Therapy Experience.
Lomash RM, Shchelochkov O, Chandler RJ, Venditti CP, Pariser AR, Ottinger EA; NIH PaVe-GT Team. Lomash RM, et al. Among authors: pariser ar. Hum Gene Ther. 2023 Mar;34(5-6):217-227. doi: 10.1089/hum.2022.232. Hum Gene Ther. 2023. PMID: 36694456 Free PMC article.
A framework for the investigation of rare genetic disorders in neuropsychiatry.
Sanders SJ, Sahin M, Hostyk J, Thurm A, Jacquemont S, Avillach P, Douard E, Martin CL, Modi ME, Moreno-De-Luca A, Raznahan A, Anticevic A, Dolmetsch R, Feng G, Geschwind DH, Glahn DC, Goldstein DB, Ledbetter DH, Mulle JG, Pasca SP, Samaco R, Sebat J, Pariser A, Lehner T, Gur RE, Bearden CE. Sanders SJ, et al. Among authors: pariser a. Nat Med. 2019 Oct;25(10):1477-1487. doi: 10.1038/s41591-019-0581-5. Epub 2019 Sep 23. Nat Med. 2019. PMID: 31548702 Free PMC article. Review.
Global Regulatory and Public Health Initiatives to Advance Pediatric Drug Development for Rare Diseases.
Epps C, Bax R, Croker A, Green D, Gropman A, Klein AV, Landry H, Pariser A, Rosenman M, Sakiyama M, Sato J, Sen K, Stone M, Takeuchi F, Davis JM. Epps C, et al. Among authors: pariser a. Ther Innov Regul Sci. 2022 Nov;56(6):964-975. doi: 10.1007/s43441-022-00409-w. Epub 2022 Apr 26. Ther Innov Regul Sci. 2022. PMID: 35471559 Free PMC article. Review.
57 results