Title: | Discontinuation of enzyme replacement therapy in adults with Pompe disease: Evaluating the European POmpe Consortium stop criteria |
Journal : | Neuromuscular disorders : NMD |
Authors: | van Kooten HA ; Harlaar L ; van der Beek NAME ; van Doorn PA ; van der Ploeg AT ; Brusse E |
Material Type: | Article |
Publisher: | England, 11/2019 |
Languages: | English |
Keywords : | adulthood ; cohort studies ; enzymotherapy ; Europe (geography) ; guidelines ; hereditary metabolic myopathy ; muscle glycogen storage disease ; Netherlands ; neuromuscular disease ; Pompe disease ; prospective studies ; therapeutic strategy |
Keywords: | adult patients;enzyme replacement therapy;Pompe disease;start and stop criteria;treatment discontinuation;treatment guidelines; |
Pubmed / DOI : | Pubmed : 31911071 / DOI : 10.1016/j.nmd.2019.11.007 |
N° Profil MNM : | 2020012 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/31911071 |
See also : |