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Table representation of search results timeline featuring number of search results per year.

Year Number of Results
2010 3
2011 3
2012 3
2014 2
2015 2
2016 3
2017 1
2018 2
2020 1
2021 1
2023 2
2024 0

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20 results

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Page 1
Research challenges in central nervous system manifestations of inborn errors of metabolism.
Dickson PI, Pariser AR, Groft SC, Ishihara RW, McNeil DE, Tagle D, Griebel DJ, Kaler SG, Mink JW, Shapiro EG, Bjoraker KJ, Krivitzky L, Provenzale JM, Gropman A, Orchard P, Raymond G, Cohen BH, Steiner RD, Goldkind SF, Nelson RM, Kakkis E, Patterson MC. Dickson PI, et al. Among authors: pariser ar. Mol Genet Metab. 2011 Mar;102(3):326-38. doi: 10.1016/j.ymgme.2010.11.164. Epub 2010 Dec 2. Mol Genet Metab. 2011. PMID: 21176882 Free PMC article. Review.
Immune response to enzyme replacement therapies in lysosomal storage diseases and the role of immune tolerance induction.
Kishnani PS, Dickson PI, Muldowney L, Lee JJ, Rosenberg A, Abichandani R, Bluestone JA, Burton BK, Dewey M, Freitas A, Gavin D, Griebel D, Hogan M, Holland S, Tanpaiboon P, Turka LA, Utz JJ, Wang YM, Whitley CB, Kazi ZB, Pariser AR. Kishnani PS, et al. Among authors: pariser ar. Mol Genet Metab. 2016 Feb;117(2):66-83. doi: 10.1016/j.ymgme.2015.11.001. Epub 2015 Nov 10. Mol Genet Metab. 2016. PMID: 26597321 Review.
Towards the international interoperability of clinical research networks for rare diseases: recommendations from the IRDiRC Task Force.
Nabbout R, Zanello G, Baker D, Black L, Brambilla I, Buske OJ, Conklin LS, Davies EH, Julkowska D, Kim Y, Klopstock T, Nakamura H, Nielsen KG, Pariser AR, Pastor JC, Scarpa M, Smith M, Taruscio D, Groft S. Nabbout R, et al. Among authors: pariser ar. Orphanet J Rare Dis. 2023 May 9;18(1):109. doi: 10.1186/s13023-023-02650-4. Orphanet J Rare Dis. 2023. PMID: 37161573 Free PMC article.
Accelerating orphan drug development.
Coté TR, Xu K, Pariser AR. Coté TR, et al. Among authors: pariser ar. Nat Rev Drug Discov. 2010 Dec;9(12):901-2. doi: 10.1038/nrd3340. Nat Rev Drug Discov. 2010. PMID: 21119719
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.
Clinical pharmacology as a cornerstone of orphan drug development.
Bashaw ED, Huang SM, Coté TR, Pariser AR, Garnett CE, Burckart G, Zhang L, Men AY, Le CD, Charlab R, Gobburu JV, Lesko LJ. Bashaw ED, et al. Among authors: pariser ar. Nat Rev Drug Discov. 2011 Oct 31;10(11):795-6. doi: 10.1038/nrd3595. Nat Rev Drug Discov. 2011. PMID: 22037026
20 results