It’s Official!

This morning Neuren released a statement in regards to Orphan Drug Status for Trofinetide (NNZ 2566)

See their announcement here.

They’ve also come out with an awesome video about the science behind Trofinetide. Take a look! Great graphics and pretty easily understood laymen terms. Video

One statement really stood out for me.. they’re applying to the European counterpart of the FDA!! To me this will really give hope to those families outside of the US, that they won’t have to come here, nor that the treatment would be out of reach for them. I hope that’s what it means.

I want to thank Rettsyndrome.org,  all it’s contributors, scientists, grant receivers, every person working behind the scenes and in front of them  that helped to make this happen. Dr. Kaminsky, you ROCK! Check this page out to see how committed Rett syndrome.org is and see Dr. Kaminsky in action!

I want to thank the US Army for having the foresight to give this medication a chance, for funding trials, and for working hard to spare our men and women protecting our country the brutal life that TBI brings. See their trial program: Intrepid 2566

I want to thank the other participants and their families for being so brave and those that supported us and hoped along side us and helped us raise funds and supported trial participants like Colleen English from Rettland.org

And I want to thank the team at Neuren, it’s directors and scientists and the stock holders. All who have  put their hard earned  time and money into a venture without guarantee.

Katie and I are in your debt.

I know this is just one more step, but it’s a STEP, not a stumble, not a backslide. It’s an onward movement to a goal we are hoping for. I try hard not to be too hopeful for Breakthrough Designation, the chances aren’t fantastic if you go by FDA numbers….only 30% of applicants get it, but I THINK the chances are better than that.

So…two things,

First, because of the video, I now know how to say Trofinetide, and it DOES roll off the tongue

and even though it’s a bit late…Happy Valentine’s Day Neuren. You are definitely in our hearts to stay.

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This entry was posted in FDA, Neuren Pharmaceuticals, NNZ-2566, Orpan Drug Status, Trofinetide and tagged , , , , , , . Bookmark the permalink.

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