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An immune tolerance approach using transient low-dose methotrexate in the ERT-naïve setting of patients treated with a therapeutic protein: experience in infantile-onset Pompe disease.
Kazi ZB, Desai AK, Troxler RB, Kronn D, Packman S, Sabbadini M, Rizzo WB, Scherer K, Abdul-Rahman O, Tanpaiboon P, Nampoothiri S, Gupta N, Feigenbaum A, Niyazov DM, Sherry L, Segel R, McVie-Wylie A, Sung C, Joseph AM, Richards S, Kishnani PS. Kazi ZB, et al. Among authors: richards s. Genet Med. 2019 Apr;21(4):887-895. doi: 10.1038/s41436-018-0270-7. Epub 2018 Sep 14. Genet Med. 2019. PMID: 30214072 Free PMC article.
Response to de Vries et al.
Herbert M, Kazi ZB, Richards S, Rosenberg AS, Kishnani PS. Herbert M, et al. Among authors: richards s. Genet Med. 2017 Nov;19(11):1281-1282. doi: 10.1038/gim.2017.48. Epub 2017 May 11. Genet Med. 2017. PMID: 28492534 Free article. No abstract available.
Atypical immunologic response in a patient with CRIM-negative Pompe disease.
Abbott MA, Prater SN, Banugaria SG, Richards SM, Young SP, Rosenberg AS, Kishnani PS. Abbott MA, et al. Among authors: richards sm. Mol Genet Metab. 2011 Dec;104(4):583-6. doi: 10.1016/j.ymgme.2011.08.003. Epub 2011 Aug 11. Mol Genet Metab. 2011. PMID: 21889385 Free PMC article.
Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease.
Kishnani PS, Corzo D, Nicolino M, Byrne B, Mandel H, Hwu WL, Leslie N, Levine J, Spencer C, McDonald M, Li J, Dumontier J, Halberthal M, Chien YH, Hopkin R, Vijayaraghavan S, Gruskin D, Bartholomew D, van der Ploeg A, Clancy JP, Parini R, Morin G, Beck M, De la Gastine GS, Jokic M, Thurberg B, Richards S, Bali D, Davison M, Worden MA, Chen YT, Wraith JE. Kishnani PS, et al. Among authors: richards s. Neurology. 2007 Jan 9;68(2):99-109. doi: 10.1212/01.wnl.0000251268.41188.04. Epub 2006 Dec 6. Neurology. 2007. PMID: 17151339 Clinical Trial.
Early treatment with alglucosidase alpha prolongs long-term survival of infants with Pompe disease.
Kishnani PS, Corzo D, Leslie ND, Gruskin D, Van der Ploeg A, Clancy JP, Parini R, Morin G, Beck M, Bauer MS, Jokic M, Tsai CE, Tsai BW, Morgan C, O'Meara T, Richards S, Tsao EC, Mandel H. Kishnani PS, et al. Among authors: richards s. Pediatr Res. 2009 Sep;66(3):329-35. doi: 10.1203/PDR.0b013e3181b24e94. Pediatr Res. 2009. PMID: 19542901 Free PMC article. Clinical Trial.
1,900 results