POM-001 phase 1/2 study of BMN 701, GILT-tagged recombinant human (rh) GAA in late-onset Pompe disease: Initial experience in 22 patients
Byrne, Barry, Barohn, Richard, Barshop, Bruce, Bratkovic, Drago, Desnuelle, Claude, Geberhiwot, Tarekegn, Hughes, Derralynn, Laforet, Pascal, Mengel, Eugen, Roberts, Mark, Lang, William, LeBowitz, JonVolume:
108
Language:
english
Journal:
Molecular Genetics and Metabolism
DOI:
10.1016/j.ymgme.2012.11.048
Date:
February, 2013
File:
PDF, 62 KB
english, 2013