Titre : | Earlier and higher dosing of alglucosidase alfa improve outcomes in patients with infantile-onset Pompe disease: Evidence from real-world experiences |
Revue : | Molecular genetics and metabolism reports, 23 |
Auteurs : | Chien YH ; Tsai WH ; Chang CL ; Chiu PC ; Chou YY ; Tsai FJ ; Wong SL ; Lee NC ; Hwu WL |
Type de document : | Article |
Année de publication : | 2020 |
Langues: | Anglais |
Mots-clés: | CK, creatine kinase;CRIM, cross-reactive immunological material;dosage;ERT, enzyme replacement therapy;early treatment;enzyme replacement therapy;GAA, acid alpha-glucosidase;GMFM, Gross Motor Function Measure;IOPD, infantile-onset PD;ITI, immune tolerance induction;NBS, newborn screening;newborn screening;PD, Pompe disease;PDMS-2, Peabody Developmental Motor Scale, Second Edition;Pompe disease;uGlc4, urine glucose tetrasaccharide; |
Pubmed / DOI : | Pubmed : 32373469 / DOI : 10.1016/j.ymgmr.2020.100591 |
N° Profil MNM : | 2020051 |
En ligne : | http://www.ncbi.nlm.nih.gov/pubmed/32373469 |