Title: | Respiratory function during enzyme replacement therapy in late-onset Pompe disease: longitudinal course, prognostic factors, and the impact of time from diagnosis to treatment start |
Journal : | Journal of neurology |
Authors: | Stockton DW ; Kishnani P ; van der Ploeg A ; Llerena J Jr ; Boentert M ; Roberts M ; Byrne BJ ; Araujo R ; Maruti SS ; Thibault N ; Verhulst K ; Berger KI |
Material Type: | Article |
Publisher: | Germany, 06/2020 |
Languages: | English |
Keywords : | enzymology ; late-onset Pompe disease ; loongitudinal study ; patient registers ; respiration (macroscopic) |
Keywords: | alglucosidase alfa;enzyme replacement therapy;late-onset Pompe disease;Pompe disease;registry;respiratory function; |
Lien associé : | NCT00231400 |
Pubmed / DOI : | Pubmed : 32524257 / DOI : 10.1007/s00415-020-09936-8 |
N° Profil MNM : | 2020061 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/32524257 |
See also : |