The Next Steps Through the Desert


Wow. While time has been passing, Neuren has quietly, behind the scenes,  been taking step after step and gaining ground with Trofinetide. Check out their latest announcements…

So, to me it feels like we went from full stop after Breakthrough Designation was denied to taking a giant mile leap forward! But, I want to thank those committed people who have been ticking off those smaller steps that got us all here.

It’s not easy waiting, I’d like an update every day… How close are we now? And today? Are we closer today? But, I imagine that would eventually drive me and everyone else crazy, lol. But, I am so grateful for these amazing updates and they were worth the wait.

I feel like things are falling into place-

Rett syndrome is listed as a medical condition eligible for defense funding for its possible connection to TBI

Orphan Drug Status in both the States and Europe

Only a small short pediatric trial followed by (possibly) only one pivitol phase III.

Clearly an open dialogue and committment by the FDA to assist in getting everything done in the right order.

I’m feeling there are not so many more hills to climb on weary feet, on hands and knees, on bellies like a man lost in a desert dragging himself to a mirage for a nonexistant sip of water. Yes, not so much more time left in this desert of no treatment, we’ll be walking out soon.


This entry was posted in Congress, cure, Neuren Pharmaceuticals, Orpan Drug Status, Rett Research, Rett Syndrome,, Trail to a Texas Trial, Trofinetide and tagged , , , , , , . Bookmark the permalink.

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