I wonder what the other 4 are? Plus a new MyoAAV benefit...? | SRPT Message Board Posts


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Msg  14465 of 14479  at  8/10/2022 9:21:03 PM  by

schmiggins


 In response to msg 14464 by  schmiggins
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Re: I wonder what the other 4 are? Plus a new MyoAAV benefit...?

If that turns out to be the case, we'd then effectively double - over the years - the "initial blast" of kids who will soon be getting the 9001 shot worldwide (likely with Roche's help again). Because the 9001 kids may eventually choose to get that MyoAAV "booster" after x number of years pass from their 9001 shot.

But there's more. If this MyoAAV version pans out, it will be at a much lower dose and we can charge way less. And because of that we can expand the number of kids worldwide who can afford it. Kids who may have been taking our monthly PPMOs in the meantime.

Possibly both the MyoAAV one shot and the various PPMOs will come on the market at the same time and they can choose. More likely that the PPMOs would be there first, I think.

At any rate, the kids would probably end up taking a mix of the GT and the RNAs - as long as the price is low enough and availability is there for both options - as it should be in time.

Whatever the case, we will be coming to these DMD kids in 3 ways and that's pretty wild after all we (and they!) have been through!

So, a huge sigh of relief should be coming for all of us long-sufferers. All's well that ends well, the poet hath said.


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Msg # Subject Author Recs Date Posted
14466 Re: I wonder what the other 4 are? Plus a new MyoAAV benefit...? schmiggins 0 8/10/2022 9:24:48 PM




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