Here is the message I tried recently to post at the Advisory Committee Group:

I couldn't find a way to comment on the news on the website, which would be a useful feature for users as well as the staff who write the news.

This message is about the September 18 recap regarding the Duchenne platform trial community meeting.
I am in favor of all the options put forward, but I am more than surprised that you didn't address the Becker patients as well.

Becker is really part of the Duchenne community, and because its onset starts later it was discovered a century later, but this discrimination should not keep going on.
There is a dearth of clinical trials specific to Becker, but on top of that Becker is systematically excluded from Duchenne trials.

This logic doesn't make any sense to me, as Becker is possibly easier to treat than Duchenne, because the lack of dystrophin is less acute on the body.
Therefore Becker patients should be part of the broader inclusion criteria, especially that the new Duchenne treatments "transform" the population to the milder variety, Becker.

So not addressing in one scoop the Becker population, will leave out the Duchenne population with a still dysfunctional dystrophin.
I appreciate all your work, but please don't leave this part of the community on the side of the road.

I hope to read back from you.

Best regards.

Views: 48

Need help using this community site? Visit Ning's Help Page.



© 2021   Created by PPMD.   Powered by

Badges  |  Report an Issue  |  Privacy Policy  |  Terms of Service