Biotech Values

[turtlepower]

Problem is a lot of the late onset don't even know they have the disease much less are seeing a genetist. I think I saw an episode on Mystery Diagnosis once with Fabry patient. Was diagnossed in 30's or 40's had noticed some signs early in life (lack of sweat) forget what but probably not till kidney problems got more troublesome and then from doc to doc.

Good point. So the problem is diagnosing fabry patients.

They aren't exactly enrolling hundreds of patients. Patients stop any therapy (even ERT) for many reasons with ERT could be AE's or costs (you often get free care in trials and even if ERT paid for could have other costs/copays)

After about a 2 year duration the 011 trial had screened 140 patients as of July 2011. I think a breakdown of how many of these patients were naive vs discontinued would be interesting. That would tell how much demand there is from discontinued patients. According to Amicus they only screened selective patients though i'm not sure what they meant by that.

http://ir.amicustherapeutics.com/releasedetail.cfm?ReleaseID=648049