Title: | 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 |
Journal : | Genetics in medicine : official journal of the American College of Medical Genetics |
Authors: | Kazi ZB, Author ; 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 |
Material Type: | Article |
Publication Date: | 14/09/2019 |
Languages: | English |
Keywords: | Pompe disease;alglucosidase alfa;antidrug antibodies;methotrexate;prophylactic immune tolerance induction; |
Pubmed / DOI : | DOI : 10.1038/s41436-018-0270-7 / Pubmed : 30214072 |
N° Profil MNM : | 2018092 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/30214072 |