Preliminary patient-reported outcomes and safety of advanced and targeted acid α-glucosidase (AT-GAA (ATB200/AT2221) in patients with Pompe disease from the ATB200-02 trial
Schoser, Benedikt, Bratkovic, Drago, Byrne, Barry J., Clemens, Paula R., Goker-Alpan, Ozlem, Kishnani, Priya, Ming, Xue, Roberts, Mark, Schwenkreis, Peter, Sivakumar, Kumaraswamy, Wright, Jacquelyn, SVolume:
126
Language:
english
Journal:
Molecular Genetics and Metabolism
DOI:
10.1016/j.ymgme.2018.12.340
Date:
February, 2019
File:
PDF, 65 KB
english, 2019