ERT positively alters the natural course of Pompe disease in adult patients

Category: Archive
September 30, 2012

This is the conclusion of a study following the response of 69 adult patients to enzyme replacement therapy (ERT) over a median of 23 months. The authors from Erasmus Medical Center in Rotterdam, The Netherlands, state that “muscle strength [is] increased and upright forced vital capacity stabilized. Functional outcome is probably best when ERT intervention is timely”.

Good to read these results in a scientific paper after the hot discussions about reimbursement of ERT in The Netherlands during the past weeks.

Source: Orphanet J Rare Dis. 2012 Sep 26;7(1):73. [Epub ahead of print]

Update 5th October 2012: The article is publicly available on the web (http://www.ojrd.com/content/7/1/73) and for download (http://www.ojrd.com/content/pdf/1750-1172-7-73.pdf).

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