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Items: 23

1.

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.

Nat Med. 2019 Oct;25(10):1477-1487. doi: 10.1038/s41591-019-0581-5. Epub 2019 Sep 23. Review.

PMID:
31548702
2.

Utilization, duration, and outcomes of neoadjuvant endocrine therapy in the United States.

Pariser AC, Sedghi T, Soulos PR, Killelea B, Gross CP, Mougalian SS.

Breast Cancer Res Treat. 2019 Nov;178(2):419-426. doi: 10.1007/s10549-019-05397-4. Epub 2019 Aug 10.

PMID:
31401686
3.
4.

Understanding the evolving phenotype of vascular complications in telomere biology disorders.

Higgs C, Crow YJ, Adams DM, Chang E, Hayes D Jr, Herbig U, Huang JN, Himes R, Jajoo K, Johnson FB, Reynolds SD, Yonekawa Y, Armanios M, Boulad F, DiNardo CD, Dufour C, Goldman FD, Khan S, Kratz C, Myers KC, Raghu G, Alter BP, Aubert G, Bhala S, Cowen EW, Dror Y, El-Youssef M, Friedman B, Giri N, Helms Guba L, Khincha PP, Lin TF, Longhurst H, McReynolds LJ, Nelson A, Olson T, Pariser A, Perona R, Sasa G, Schratz K, Simonetto DA, Townsley D, Walsh M, Stevens K, Agarwal S, Bertuch AA, Savage SA; Clinical Care Consortium for Telomere-associated Ailments (CCCTAA).

Angiogenesis. 2019 Feb;22(1):95-102. doi: 10.1007/s10456-018-9640-7. Epub 2018 Aug 25.

PMID:
30168024
5.

Representation of Women and Minorities in Clinical Trials for New Molecular Entities and Original Therapeutic Biologics Approved by FDA CDER from 2013 to 2015.

Chen A, Wright H, Itana H, Elahi M, Igun A, Soon G, Pariser AR, Fadiran EO.

J Womens Health (Larchmt). 2018 Apr;27(4):418-429. doi: 10.1089/jwh.2016.6272. Epub 2017 Oct 19.

PMID:
29048983
6.

A role for plasma cell targeting agents in immune tolerance induction in autoimmune disease and antibody responses to therapeutic proteins.

Rosenberg AS, Pariser AR, Diamond B, Yao L, Turka LA, Lacana E, Kishnani PS.

Clin Immunol. 2016 Apr;165:55-9. doi: 10.1016/j.clim.2016.02.009. Epub 2016 Feb 27. Review.

PMID:
26928739
7.

Investigational New Drug applications: a 1-year pilot study on rates and reasons for clinical hold.

Lapteva L, Pariser AR.

J Investig Med. 2016 Feb;64(2):376-82. doi: 10.1136/jim-2015-000010.

PMID:
26911627
8.

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.

Mol Genet Metab. 2016 Feb;117(2):66-83. doi: 10.1016/j.ymgme.2015.11.001. Epub 2015 Nov 10. Review.

PMID:
26597321
9.
10.

Important role of translational science in rare disease innovation, discovery, and drug development.

Pariser AR, Gahl WA.

J Gen Intern Med. 2014 Aug;29 Suppl 3:S804-7. doi: 10.1007/s11606-014-2881-2.

11.

Phenylketonuria Scientific Review Conference: state of the science and future research needs.

Camp KM, Parisi MA, Acosta PB, Berry GT, Bilder DA, Blau N, Bodamer OA, Brosco JP, Brown CS, Burlina AB, Burton BK, Chang CS, Coates PM, Cunningham AC, Dobrowolski SF, Ferguson JH, Franklin TD, Frazier DM, Grange DK, Greene CL, Groft SC, Harding CO, Howell RR, Huntington KL, Hyatt-Knorr HD, Jevaji IP, Levy HL, Lichter-Konecki U, Lindegren ML, Lloyd-Puryear MA, Matalon K, MacDonald A, McPheeters ML, Mitchell JJ, Mofidi S, Moseley KD, Mueller CM, Mulberg AE, Nerurkar LS, Ogata BN, Pariser AR, Prasad S, Pridjian G, Rasmussen SA, Reddy UM, Rohr FJ, Singh RH, Sirrs SM, Stremer SE, Tagle DA, Thompson SM, Urv TK, Utz JR, van Spronsen F, Vockley J, Waisbren SE, Weglicki LS, White DA, Whitley CB, Wilfond BS, Yannicelli S, Young JM.

Mol Genet Metab. 2014 Jun;112(2):87-122. doi: 10.1016/j.ymgme.2014.02.013. Epub 2014 Mar 6.

PMID:
24667081
12.

Cross-sector sponsorship of research in eosinophilic esophagitis: a collaborative model for rational drug development in rare diseases.

Fiorentino R, Liu G, Pariser AR, Mulberg AE.

J Allergy Clin Immunol. 2012 Sep;130(3):613-6. doi: 10.1016/j.jaci.2012.07.011. Epub 2012 Jul 31.

PMID:
22857796
13.

Rare cancer trial design: lessons from FDA approvals.

Gaddipati H, Liu K, Pariser A, Pazdur R.

Clin Cancer Res. 2012 Oct 1;18(19):5172-8. doi: 10.1158/1078-0432.CCR-12-1135. Epub 2012 Jun 20.

14.

Characteristics of rare disease marketing applications associated with FDA product approvals 2006-2010.

Pariser AR, Slack DJ, Bauer LJ, Warner CA, Tracy LA.

Drug Discov Today. 2012 Aug;17(15-16):898-904. doi: 10.1016/j.drudis.2012.04.011. Epub 2012 May 5.

PMID:
22564486
15.

The role of immune tolerance induction in restoration of the efficacy of ERT in Pompe disease.

Lacaná E, Yao LP, Pariser AR, Rosenberg AS.

Am J Med Genet C Semin Med Genet. 2012 Feb 15;160C(1):30-9. doi: 10.1002/ajmg.c.31316. Epub 2012 Jan 17.

PMID:
22253234
16.

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.

Nat Rev Drug Discov. 2011 Oct 31;10(11):795-6. doi: 10.1038/nrd3595.

PMID:
22037026
17.
18.

Training the translational scientist.

Jackson RD, Gabriel S, Pariser A, Feig P.

Sci Transl Med. 2010 Dec 22;2(63):63mr2. doi: 10.1126/scitranslmed.3001632.

PMID:
21178132
19.

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.

Mol Genet Metab. 2011 Mar;102(3):326-38. doi: 10.1016/j.ymgme.2010.11.164. Epub 2010 Dec 2. Review.

20.

Accelerating orphan drug development.

Coté TR, Xu K, Pariser AR.

Nat Rev Drug Discov. 2010 Dec;9(12):901-2. doi: 10.1038/nrd3340.

PMID:
21119719
21.

Therapies for inborn errors of metabolism: what has the orphan drug act delivered?

Talele SS, Xu K, Pariser AR, Braun MM, Farag-El-Massah S, Phillips MI, Thompson BH, Coté TR.

Pediatrics. 2010 Jul;126(1):101-6. doi: 10.1542/peds.2009-3246. Epub 2010 Jun 21.

PMID:
20566615
22.

Oxidant-induced apoptosis is mediated by oxidation of the actin-regulatory protein cofilin.

Klamt F, Zdanov S, Levine RL, Pariser A, Zhang Y, Zhang B, Yu LR, Veenstra TD, Shacter E.

Nat Cell Biol. 2009 Oct;11(10):1241-6. doi: 10.1038/ncb1968. Epub 2009 Sep 6. Erratum in: Nat Cell Biol. 2009 Nov;11(11)1387.

23.

Neutralizing antibodies to therapeutic enzymes: considerations for testing, prevention and treatment.

Wang J, Lozier J, Johnson G, Kirshner S, Verthelyi D, Pariser A, Shores E, Rosenberg A.

Nat Biotechnol. 2008 Aug;26(8):901-8. doi: 10.1038/nbt.1484.

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