Replies to post #149920 on Biotech Values

[jq1234]

That might be the case. As it is now, higher dystrophin increase from baseline from 30mg/kg didn't translate into 6MWT. Of course we don't know whether this was due to lower baseline value.

[zipjet]

SRPT

I think the important observation about dystrophin production would be if the protein is actually incorporated into the costameres. Only then is it functionally meaningful.

Thank you.

Increased walk distance infers that it is functionally meaningful. Anecdotal reports also suggest it is meaningful.

In case you are not familiar with MDM males, they are treated with steroids and rapidly become obese even morbidly obese. Because of that, I place more weight on the increased walking rate/distance.

MDM is a disease begging for treatment.

I wish the n was 80 rather than 8. But given the latter, the drug looks to me like it is still 3 years to approval.

ij