G.O.20 - Reveglucosidase alfa (BMN 701), a GILT-tagged recombinant human acid alpha glucosidase (rhGAA), evaluation in late-onset Pompe disease: Preliminary clinical efficacy and safety results of an extension study (72-week results)

Autor: Hiwot, T. *, Barohn, R., Bratkovic, D., Byrne, B., Desnuelle, C., Hughes, D., Laforêt, P., Mengel, E., Roberts, M., Yang, K., Heusner, C., Dummer, W.
Zdroj: In Neuromuscular Disorders October 2015 25 Supplement 2:S313-S314
Databáze: ScienceDirect
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