Title: | Long-Term Observation of the Safety and Effectiveness of Enzyme Replacement Therapy in Japanese Patients with Pompe Disease: Results From the Post-marketing Surveillance |
Journal : | Neurology and therapy, 8, 2 |
Authors: | Nagura H, Author ; Hokugo J ; Ueda K |
Material Type: | Article |
Publisher: | New Zealand, 2019 |
Languages: | English |
Keywords : | drug monitoring ; enzymotherapy ; muscle glycogen storage disease ; neuromuscular disease ; observational research ; Pompe disease ; therapeutic efficiency |
Keywords: | alglucosidase alfa;clinical outcome;Enzyme replacement therapy;japanese cohort;Pompe disease;post-marketing surveillance; |
Pubmed / DOI : | Pubmed : 31559584 / DOI : 10.1007/s40120-019-00157-4 |
N° Profil MNM : | 2019093 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/31559584 |