$SRPT
So far 100% no drug related adverse events:
4 boys 50 mg/kg 52 weeks
2 boys 50 mg/kg 30 weeks
4 boys 30 mg/kg 52 weeks
2 boys 30 mg/kg 30 weeks
19 boys at does between .5 and 20 mg/kg for 12 weeks.

Is there a drug out there that doesn't have adverse events.

Probably 100% of the 12 boys will have increased dystrophin, this is the surrogate endpoint.

All the boys have to do is maintain function or more slowly lose function, not gain function for the drug to be a raging success.

It appears that at least 2 boys have been gaining functionally.

Why wouldn't the FDA improve a drug that helped only 25% of the boys, if it harmed no one? And this is a drug that at 32 and 36 weeks proved to statistically help all (?) the boys on the 50 mg/kg dose and 75% of the boys if you combined the 30 and 50 mg/kg cohorts.

Seems conditional approval and a followup confirmatory trial is the least harmful thing the FDA could do.

In the USA for the lack of skipping exon 51, 90 boys die each year, another 90 lose the ability to walk, another 90 lose the ability to pick up a glass of water.

The competitive drug has proven to have some toxicities.

Now a concern, it there will be a lot of other measures besides 6 MWT and dystrophin to be presented to the FDA, none of it has been shared with investors. It is hard to imagine a lot of it won't be presented in Perth.

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