PPMD Community

PLEASE SIGNT FDA SAY YES PETITION AND ENCOURAGE OTHERS TO DO IT

Besides signing the petition, please ask your spouse, adult kids, friends, co workers, or others to sign the petition.  Post it on Facebook, Twitter, Linked In, other social networks.

https://petitions.whitehouse.gov/petition/urge-fda-say-yes-accelera...

Every year this approval is delayed about 90 boys in the USA will die from DMD that could be saved by skipping exon 51. Another 90 will take their last step, and another 90 will take their last unassisted sip of water.

Approving Eteplirsen will open the floodgates for skipping other exons.  It won't be instantaneous.  But it will happen sooner than it would if Eteplirsen is further delayed.  The FDA needs to recognize 6MWT and dystrophin as a biomarker

Key scientists have testified to the FDA and Congress the drug works and appears very safe.  Even, if this doesn't help your boy, it will help directly some boys with DMD and start the long road to helping many others where exon skipping technology will work.

Ideally the FDA will give this a full approval with monitoring and recognize dystrophin as a biomarker.  The FDA should provide assistance in writing the NDA as has happened for other rare diseases

.

Ask your congress people to push for approval! Ask others.  You can make a difference NOW!

Views: 327

Replies are closed for this discussion.

Replies to This Discussion

There was a very encouraging presentation this morning, showing how eteplirsen has almost completely stopped the progression of DMD in boys 7 years or older, for over 120 weeks with no reported side effects. Is it possible that a cure may be around the corner using Sarepta's research? We'll know if only the FDA would grant it some sort of approval. And many many kids could see their lives turn around. Let's 'make a difference' - get this medicine into hands who need it, soon - no more delaying in the government agency which is charged by FDASIA with bringing cures rapidly to the community.

No, a cure is not "around the corner".




Tommy Paine said:

There was a very encouraging presentation this morning, showing how eteplirsen has almost completely stopped the progression of DMD in boys 7 years or older, for over 120 weeks with no reported side effects. Is it possible that a cure may be around the corner using Sarepta's research? We'll know if only the FDA would grant it some sort of approval. And many many kids could see their lives turn around. Let's 'make a difference' - get this medicine into hands who need it, soon - no more delaying in the government agency which is charged by FDASIA with bringing cures rapidly to the community.

Keith, I apologize for the way I worded my first post. I didn't intend for it to be inconsiderate or unfeeling. I now understand it was simplistic. Clearly, even as it is providing great hope to many now, the time needed to apply this research to all DMD patients is significant; sadly so. Once again, I'm sorry for not acknowledging that in my first post, My hopes and prayers are with you.

This thread had been locked because a discussion on this topic already exists: http://community.parentprojectmd.org/forum/topics/whitehouse-petiti.... Please move the discussion there. Thank you! 

RSS

© 2017   Created by PPMD.   Powered by

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