Pompe disease patient from New Zealand

[AllysonNZ]

Hi there, just joined up. I have Pompe disease and was on a trial for a new enzyme replacement therapy for 5 years before the trial was cancelled.

[ciscrp_official Partner]

Welcome AllysonNZ ! Did you ever find out why the trial was canceled? Have you participated in any other trials since then?

[AllysonNZ in reply to ciscrp_official]

They said it was because they wanted to concentrate on drugs that would earn them $500million and more. They speculated this one would bring them "only" $400million. But it's more likely that it was because the drug wasn't well tolerated by pretty much everyone who tried it. I haven't been on a trial since that one.