Biotech Values

[iwfal]

BMRN -

the one thing I find a bit harder to sign off on is several families claiming their son would deteriorate during the period drug was suspended and then stabilize after drug was started (sometime afterward). From what I've learned in DMD that is unusual.

At least for 6MWD it isn't uncommon to drift up and down some - see, for instance, Goemanns. What would be odd is a bunch of patients stabilizing at, say, 200m for 2 years.