One Step Closer

imageIt is just after midnight and my girl is sleeping (thank goodness!!) and I wonder if her and other Rett children’s dreams just became filled with butterflies. Why butterflies? Because they are to me a most beautiful symbol of metamorphosis, of evolving, of change. Tonight, as she slept this came our way….




NNZ-2566 has been granted Orphan Drug Designation.

What does it mean? To be honest, more than I can understand. I know it means huge incentives for Neuren, with 7 years marketing exclusivity if (when!) the FDA approves it for use, tax breaks etc. but what I FEEL in my heart is that the FDA is acknowledging the Rett Community needs this, our children need hope, us parents need hope. And, I FEEL like a magician just placed a cocoon in his hand, blew in it, opened it up and a thousand butterflies filled the air…for there it is, in writing, by a FEDERAL AGENCY- “treatment of Rett Syndrome”.

The world is achanging and it’s a beautiful thing.

(Taken at the Butterfly House in Houston)

This entry was posted in FDA, Neuren, nnz 2566, Rett Syndrome, Trofinetide and tagged , , , , , , , , . Bookmark the permalink.

4 Responses to One Step Closer

  1. keli barish says:

    Hi , I’m little bit confused because it looks like it wasn’t approved per their website?

    • melelllan says:

      I was, too. 🙂 but what it means is the “designation” is approved, but the drug is not yet approved for use. STILL huge!

  2. Keli Barish says:

    Yes it is!! 😃

  3. mdfcapecod says:

    knock knock knocking on heavens door….

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