Open-label extension study following the Late-Onset Treatment Study (LOTS) of alglucosidase alfa

Article ID	Journal	Published Year	Pages	File Type
10833499	Molecular Genetics and Metabolism	2012	6 Pages	PDF

Abstract

âº This extension study continued Late-Onset Treatment Study (LOTS) out to 104Â weeks. âº Patients maintained the improved walking distance at 104Â weeks. âº Patients maintained stabilization in pulmonary function at 104Â weeks. âº Treatment-related AEs were mainly infusion-associated reactions (35% of patients). âº No deaths or anaphylactic reactions were observed.

Keywords

Inherited metabolic disease Neuromuscular disease Pompe disease Genetics Glycogenoses

Related Topics

Life Sciences Biochemistry, Genetics and Molecular Biology Biochemistry

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Open-label extension study following the Late-Onset Treatment Study (LOTS) of alglucosidase alfa

Authors

Ans T. van der Ploeg, Richard Barohn, Lisa Carlson, Joel Charrow, Paula R. Clemens, Robert J. Hopkin, Priya S. Kishnani, Pascal LaforÃªt, Claire Morgan, Sharon Nations, Alan Pestronk, Horacio Plotkin, Barry E. Rosenbloom, Katherine B. Sims, Elisa Tsao,