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genisi

11/06/12 10:01 AM

#151816 RE: ghmm #151815

BMRN

Well, they were smart to go with the 6MWD. The bone growth can be shown in a post marketing trial. Indeed it's more than a skeletal disease, damage to many organs (heart, eyes, ears, lungs, GI) and no doubt that the sooner you start ERT to restore deficiency in the enzyme is better and treatment is needed for lifetime.