Prospective exploratory muscle biopsy, imaging, and functional assessment in patients with late-onset Pompe disease treated with alglucosidase alfa: The EMBASSY Study

Autor: van der Ploeg, Ans, Carlier, Pierre G., Carlier, Robert-Yves, Kissel, John T., Schoser, Benedikt, Wenninger, Stephan, Pestronk, Alan, Barohn, Richard J., Dimachkie, Mazen M., Goker-Alpan, Ozlem, Mozaffar, Tahseen, Pena, Loren D.M., Simmons, Zachary, Straub, Volker, Guglieri, Michela, Young, Peter, Boentert, Matthias, Baudin, Pierre-Yves, Wens, Stephan, Shafi, Raheel, Bjartmar, Carl, Thurberg, Beth L.
Zdroj: In Molecular Genetics and Metabolism September-October 2016 119(1-2):115-123
Databáze: ScienceDirect