ERT for late-onset Pompe disease

Posted by & filed under Part 16: LYSOSOMAL DISORDERS, Part 25: MUSCLE.

van der Ploeg AT et al. A randomized study of alglucosidase alfa in late-onset Pompe’s disease. N Engl J Med. 2010 Apr 15;362(15):1396-406

In this study, alglucosidase alfa resulted in improved walking distance and stabilization of pulmonary function for patients with late-onset Pompe’s disease.

posted by Philippe Campeau

Leave a Reply

You must be logged in to post a comment.