About

 

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 Lancasterm_lancaster

Katelin’s mom

 

 

 

 

brianna bBrianna B.

Sibling Consultant

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9 Responses to About

  1. johncaitken says:

    Great news for Katelin and family with pediatric trial results
    Have a great day
    Best wishes from Warracknabeal, Australia

    • melelllan says:

      Thank you. We have def. not been on this journey alone, thanks to all there who held up such a shining light to follow.

  2. Tarek says:

    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,

    • melelllan says:

      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.

  3. aras says:

    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…

    • melelllan says:

      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. 🙂

      • aras says:

        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.

  4. melelllan says:

    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!

    • aras says:

      Thank you so much for the clarification. Black currants seems fine, for now. Here’s too hope! Thank you.

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