There are so many people who have helped make Katelin’s participation possible. Please follow Katelin and I as we journey into the unknown with the NNZ-2566 trial and beyond. A cure for Rett Syndrome is within our reach and hopefully by participating in this study, Katelin can be part of that cure and the girls and boys that come after her will not have to suffer the devastating effects of this disorder. Thank you to all the many, many wonderful people who are making this possible.
For more information about Rett Syndrome go to http://www.rettsyndrome.org
Staff:
Melinda Lancaster
Katelin’s mom
Brianna B.
Sibling Consultant
Trail to a Texas Trial 
Great news for Katelin and family with pediatric trial results
Have a great day
Best wishes from Warracknabeal, Australia
Thank you. We have def. not been on this journey alone, thanks to all there who held up such a shining light to follow.
Hi Melinda, sorry if I’m bothering you but as all others I do follow your posts every day to keep that hope you drawn, is the day coming to see our daughters talking, walking and living the regular life ? 24 hours I asked God this hope to come true , why not as long I belief in miracle , is Trofinetide holding our dreams and hopes ? I don’t know really but I hope and living to belief in it, again sorry for disturbing you with my question but I see that you are the right person with huge knowledge of the promising medicine named trofinetide
Regards to you and your angle katelin
nmerzain@gmail.com
Father of Nay 7 years old,
Oh, you are not bothering me! I don’t know the answer to your question. I hope so. I believe it will make a tremendous difference, but there are many unanswered questions- how much will it help, will girls respond differently, will a point be reached when there is no more improvement and there still are issues that need to be addressed. Only time will tell the answers, but I do not believe it is a false hope to think that Trofinetide may help a great deal. Keeping you in my thoughts as we wait for this next trial and keep my fingers crossed it is a success.
Dear Melinda and Katelin,
I hope that you are both fine. Wanted to share some news on trofinetide:
“GlobalData expects trofinetide to launch in the US in Q2 2021”
https://www.pharmaceutical-technology.com/comment/tetra-granted-fda-orphan-drug-designation-fxs-treatment-will-enough/
and
“Neuren Pharmaceuticals may have secured an all-important deal to get its genetic brain disorder drug out of the lab and into the market”
https://stockhead.com.au/health/neuren-may-be-about-to-do-a-5-3m-deal-for-its-orphan-brain-drug/
Do you think that these news signal that Trofinetide has a very high chance of being approved by the FDA maybe in 2020? Also do you know if a 2 year old may participate in the Phase 3 trial?
All the best…
Hi, As we just got the good news yesterday and my girl not feeling all that well, I haven’t had a chance to read up. Though I am very excited about it, it’s def. a move in the right direction. At this time, I feel it is unlikely a two year old can participate since they’ve only trialed 5-45, so my guess is that’s the age range, but really there’s no telling until the criteria comes out. I’ve promised to look into the ins and outs of this news today and hope to have personal conclusions by tomorrow. 🙂
Dear Melinda and Katelin,
I hope that both of you are fine.
Like you I am research addict, googling anything and trying to find something. Just check this out, is this REAL? It is posted on 22 May 2018:
https://www.preprints.org/manuscript/201805.0300/v1
you can also google and read the whole article
“Rett Syndrome: Treatment with IGF-1, Melatonin,
Blackcurrant Extracts, and Rehabilitation”
If this is real the treatment is out there already!
“pills by New Zealand Pharmaceuticals NZP?!”
“Currently, a chemically modified form of GPE, called Trofinetide (formerly called NNZ-2566), makes the peptide suitable for oral administration, with a longer half-life in plasma and easy passage to the brain.”
Of course its not a cure but this is great if real. Can you confirm it through the people you know?
Wish you a pleasant and hopeful day.
I have seen the article already. I hashed it out with a dad in France. 🙂 This is not a true scientific study; one child, no control etc. An IGF-1 trial was done in Boston Children’s Hospital and the results were mixed. Verbal communication by parents was also varied, though subjective, some saw improvement, others not. As I told the other dad. There is 0 harm in trying black currants, so if you feel you want to try it, go ahead. 🙂 As far as Trofinetide goes, almost this whole blog is about Trofinetide. It began as NNZ 2566 and got a name a few years ago. I am extremely optimistic about the efficacy of Trofinetide and I think all parents should be. It is not available yet; there is a third and final pivotal trial to do yet. It is slightly behind schedule, but it is coming. Most likely it will encompass ages 5-45. When they begin active recruiting for the trial, notice will come from several sources and will likely spread like wildfire over Facebook. Here’s too hope!
Thank you so much for the clarification. Black currants seems fine, for now. Here’s too hope! Thank you.
I’m gone to inform my little brother, that he should also visit this web site on regular basis
to get updated from hottest reports.
Mel 🙂
I came across your family’s story at a young age, on an investment notice board of all places. A grade 9 business class (2010 in Australia) had us find a stock and follow it for the semester. Somehow I found Neuren and an even bigger somehow for a teenage boy was the maintained interest :S
My dad helped set up a trust account for underage kids who wanted to invest and I was on the board (not literally though – peanuts holding). Over the years I’ve added and subtracted to my holding, coinciding with many of life’s ups and downs, but like that teenage boy, my interest maintained. I just hope now that it was my subconscious thinking about your daughter, your family and others battling away on life’s journey, not me thinking about myself. In saying that, the fact I studied business instead of science at university is somewhat telling 😉
Regardless, lives are on the cusp of changing for the better and I want to be on the board (literally). To coincide, I just sold my Neuren holding (still relative peanuts) and donated it all (bar a burrito bowl – fucking good one though) to the charity listed on your website. I don’t want to profit off people’s suffering. And although we don’t share one another’s sufferings, you helped spark my realisation of others back in grade 9. I’ve been working since university exclusively in not for profits and am as cynical of “good” as can be as a result, but you also sparked my belief in hope 🙂
Thank you for helping me find the meaning of good,
Seamus
Dear Seamus, this is by far one of greatest things anyone has ever said to me or about me. I hope it’s the best burrito bowl ever. Knowing that a young man can find such an interest and compassion from an assignment gives ME great hope. Thank you for your donation and I will make sure the founder knows about the man who made it. I hope you continue to follow Katie’s journey when 🤞she gets it again, people like you have made this all possible.
With heartfelt gratitude for caring,
Melinda