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biomaven0

03/30/15 10:22 PM

#189406 RE: ghmm #189402

>FGEN

The trouble with fibrosis is that the end points are all very difficult. So even if you have a good drug you will often have difficulty proving it. DMD might actually prove the easiest indication, which would be pretty ironic. Likely they can use cardiac function or FVC in older children, which are much easier measures than walking distance.

Peter