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Bourbon_on_my_cornflakes

07/04/21 11:06 PM

#319571 RE: raja48185 #319567

For Rett, we have amazing results from the first trial, should approve it on that alone on accelerated basis.

Effectiveness is clear and obvious

Remember, FDA approved Adu because no alternatives. Even though it doesn't work.

Better case here for Rett. No alternatives and people suffering. And numerous trials show 273 is very safe. And very effective.

Accelerated approval NOW!

The other trials can serve a P4s.

No excuses.

attilathehunt

07/04/21 11:56 PM

#319578 RE: raja48185 #319567

Why not expect approval on low dose and when the 2nd trial is completed then add the children and later approve the higher dose after the 3rd trial?

There is an urgent unmet need so why not approve in stages just like the trials were set up? If Excellence and Avatar were not started then what would the FDA do will the low dose trial? It should be obvious.