All is Not Quiet on the Trofinetide Front #2; A Balancing Act

* Warning* Looooong post! If you aren’t interested in financial aspects and just want an update on logistics that have affected the timeline, skip to the second half- there’s also a link to a new update from Acadia, the pharmaceutical company that will be handling the trial. 🙂

*Disclaimer* Everything in this post is my opinion, my way of making sense of the steps being taken and my interpretation [in my limited scope of understanding] of some of the financial/logistical aspects necessary to heed in getting Trofinetide to our children. Everyone should do their own research when considering stock options etc. and the ONLY purpose of this post is to try and explain aspects of getting Trofinetide to market that impacts the timeline.

I will be the first to tell you all, finances and stocks, stock prices, the balance companies need to strike to meet their shareholders’ expectations vs getting new products approved, whatever they may be, just goes right over my head. I want to interject here… Without stockholders there would NEVER have been this blog in the first place because there would be no Trofinetide, so I hope they make a killing. I do know, however, that these expectations are an integral part of how Trofinetide is making its way to market. No company could just plow ahead, throwing money into trials with nary a thought to the long term.

I liken this to…say, a small independent publisher with limited resources- right out of the gate they burn up all their funds publishing a multitude of books, paying illustrators and  printing costs and then find out they have no money left for marketing. The end result? Limited sales and small return on their investment, leading to a failure to thrive in the world of mega-publishers. Even I can see that’s bad business.

So, what we’ve got here is Neuren all the way over in Australia and they know that North America, specifically the United States, is the target to achieve the best chance at Trofinetide getting to market. It began nearly 15 years ago with Intrepid-2566, a program funded by the US Army in conjunction with Walter Reed Army Hospital to address traumatic brain injuries (TBI) in soldiers. This is an interesting story all on its own and I encourage anyone interested to look it up. I’m emotionally invested in finding a way to improve mortality due to TBI as my best friend CPO Theodore Moreland, USN SEAL, SEAL Team 2, died from a TBI sustained in a training accident. If my child did not have Rett syndrome, this whole blog would still probably have started with Trofinetide, just Intrepid-2566 instead of the Texas trial. But, I digress.

Let’s just jump to the present because that’s where all our focus is. I read every announcement Neuren puts out (the stock ones I try to make sense of, but so far I can’t make hide nor hair of any of those, lol). It is mind boggling all the steps it takes to keep this balance because if the scale tips it most certainly will tip to the wrong side. Like it or not, funding a trial takes a LOT, lots and lots, of money, staggering amounts. There was no way Neuren could do it on their own; if we had to wait until they had enough funding on their own we wouldn’t be looking at a pivotal trial starting this year, probably not even in this decade. To this end, Neuren partnered with a US company-Acadia Pharmaceuticals.

This partnership came with all kinds of incentives for both companies. There are a few I understand.

  • Acadia funds the trial (upwards of 50-60 millions of dollars) and gets the North American rights to Trofinetide and first dibs on the rights in Europe and rest of the world.
  • Neuren gets this trial funded, keeps all rights outside of North America AND gets access to all the data from the trial so they don’t have to repeat it. They also got a significant down-payment of $10,000,000
  • Acadia gets the North American rights to Trofinetide in Fragile X, another devastating genetic disease which occurs mostly in males. There has been one Phase II trial so far. In the case of Fragile X, there are too many dendrites on the nerve cells causing sensory overload among other issues. Trofinetide takes the extra ones away, normalizing the number and length just as it does in Rett syndrome. (Isn’t science grand!?!)
  • Neuren keeps the rights for Fragile X outside of North America with similar conditions as they have with Rett syndrome and Acadia.
  • Neuren gets 1/3 of revenue from any “Pediatric Voucher” granted by the FDA. This is a program that grants an expedited marketing review (from 10 months to 6 months)-speeding up the time to market. Because Trofinetide already has Fast Track and Orphan Drug status, it automatically has an expedited review, so this can be sold to another company. It can go for $100,000,000!!!

That’s all about finance I understand. It’s not much, I know, but this kind of deal took a lot of brokering. I do know, having met the team at Acadia in San Diego, that I personally have complete confidence in having them on our side; clearly Neuren feels the same. There are three resources I can recommend to those who want more insight into this deal. The first is Hotcopper, the Australian stock exchange forum; the second is Neuren’s website and all of its announcements; and this podcast with Dr. Richard Treagus, Executive Chairman.

I will also include this link from Pitt Street Research that goes over all things Neuren in regards to stock pricing etc.


Now, on to some logistics of a Phase III Trofinetide trial that have impacted the timeline. Here, again, the FDA has played a major part. The FDA dictates the standards for any trial in America-from length, number of participants, to the amount of stock a pharmaceutical company has to have in order to complete the trial within the guidelines decided upon. Enter Acadia! Here is a nice overview on this partnership-AusBiotechInvest and Partnering 2018 

First and foremost, no matter what anyone may hear from their Rett specialist, the Phase III has not been “held up” because of the “drug company”; No Acadia=no trial, it’s as simple as that.

As discussed in the previous blog post, the manufacturing enough trial medication is the largest hurdle that needs to be jumped over. It’s not just making Trofinetide; it’s making it storable, making a multiple dose compound so parents aren’t given a hundred tiny little bottles; it’s making doses in a variety of strengths based on a four-tiered system governed by the weight-classes of participants. It even had to take into account that some people are allergic to strawberry which is the original flavoring and make it so that no one can be denied Trofinetide because they are allergic to strawberry; how much would that suck?!?

When I met with the San Diego Acadia team, these were the issues foremost in my mind and I was happy that there was a completely satisfactory answer for every one. The other substantial concern for parents is the cost of participating, so I’m happy to report that Acadia and will work together to help in reimbursing for reasonable travel costs associated with the trial. A shout out to our very own Colleen English who rocks Rettland in so many amazing ways. Without her vision, seeing a need and taking up the cause, the cost of participation would put trials out of reach for many.

Lastly, I think we need to take a look at and appreciate the time consuming effort that must go into choosing sites, creating standards of reporting, training personnel and all the thousand little things we, as parents, don’t even know go into this sort of thing.

Just yesterday, I received a message from Acadia and they sent along this update, shared on When you read it, you will see that they are striving to make this trial as easy as possible to participate in, within the need to adhere to strict guidelines set by the FDA. Of particular note is their hope to include even more sites than the Phase IIb (pediatric trial).

If you’ve made it to the end of this post… well done! and thank you. 🙂 Hope it’s answered some questions you may have had.



Posted in Acadia, cure, FDA, Neuren Pharmaceuticals, Orpan Drug Status, orphan drug designation, Rett Research, Rett Syndrome,,, TBI, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial, Trofinetide Phase III | Tagged , , , , , , , , , , , , , , , , , , , | 5 Comments

All is Not Quiet on the Trofinetide Front #1 A Chess Game

It’s been very quiet in the public eye in regard to Trofinetide. We all are in a holding pattern as we wait for Phase III to begin, but BEHIND the scenes things are progressing strategically and clearly, like a chess game, each move calculated for optimum impact.



What don’t we know yet about Phase III:

  • exact date for Phase III to start
  • the age cutoff
  • the criteria
  • the sites

What DO we know:

  • Phase III will start in the later half of 2019
  • Sites will only be in America
  • It will last about a year
  • Doses will be adjusted for weight in a four tiered system
  • It will include approximately 180 participants
  • will be assisting in reimbursement of travel expenses.
  • The delay in starting the Phase III trial is NOT because of the partnership with Acadia (the North American company that Neuren has partnered with to get the Phase III done and Trofinetide to market)

But, we’ve know all this for awhile.

What has BEEN happening since the Phase II wonderful, outstanding results?? And WHY is everything taking so long?? There are two major “fronts” (in my opinion) in this battle to get Trofinetide to our children- The FDA and the financial/logistical aspects. Today we’ll discuss the FDA, it’s by far more straightforward.

The FDA is a government agency. Let’s be honest, when does the government do anything as fast as we would like? Ok, that’s a little facetious, but sort of true. We do, however, have to keep in mind we damn sure want this to be done perfectly because it would really suck if at the end the FDA goes “oops, um, this part here wasn’t conducted properly” and we have to go through it all over again. To that end, Neuren and the FDA conducted an “End of Phase II Meeting” in October of 2017.  This addressed key issues of the Phase III. I know, you’re asking yourselves-that was 2 years ago! What’s taking so long then?

In June 2018, the Chairman’s Address at Neuren’s annual stockholders’ meeting went over some of the conditions the FDA placed on Neuren in order to prepare for Phase III. Two of the most time consuming conditions have been-

  • “In preparation for the Phase 3 trial, which will involve a longer duration of treatment than the Phase 2, there is a standard requirement to generate additional data from non-clinical studies in two species. We previously completed one of the studies and the second is currently in progress. The second study, which will conclude in Q4 2018 has two components and I am pleased to report that both components are on schedule, with no unexpected findings to date.”
  • Manufacturing is a key element of the preparation for Phase 3. A Phase 3 trial has to be conducted using the commercial (“to-be-marketed”) product, which means that changes to the Phase 2 product supply arrangements and a significant investment have been required for manufacturing of both the drug substance and the finished drug product liquid formulation and packaging.

It is the second condition that has really been the driving force behind how soon/late the Phase III trial starts. You can read more about it in the link above. However, we can all see by the first requirement alone, having most likely just finished, the Phase III could not have started until 2019. This data needs to be given to the FDA and has to be signed off on.

The second condition is something I will go into more in the next blog post. For new parents and for those who may need a refresher- Trofinetide is targeting the underlying biological cause of Rett syndrome and doesn’t treat any singular or particular set of symptoms. It’s had two very successful Phase II trials (one adult, one pediatric); it is well tolerated with no safety concerns identified. If you’re interested in a breakdown of the Phase IIb (pediatric) results, you can look back at my previous posts on the results. 

A quick visual for new parents who might be wondering what Trofinetide actually does.

In the brain, neurons have dendrites. Dendrites act as telephone wires. Between each one is a miniscule distance the signal has to jump, this is called the synaptic space. In the normal brain there are multiple dendrites on each side of a neuron to send out and receive instructions. In the Rett brain, the dendrites are both too few and too short. This means that while the brain is sending out the correct signals, the synaptic space is too vast and the signal gets lost in the space between.

Take your hands and spread the fingers out until they are almost touching-this is the normal neuron/dendrite with a very small synaptic space; now close your fists-this is the Rett neuron with a huge synaptic space. Now, open your hands again with the fingers almost touching-this is what Trofinetide does, regrows the dendrites to the correct number and length, allowing the brain’s signals to get to where they need to go.

I cannot end this segment without discussing Margaret Brimble, the Mother of Trofinetide, because we all owe her such a debt. Recently made a Dame, Dame Brimble has also been given the high honor of “Fellow of the Royal Society.” Without her own passion for discovering new drugs to treat a variety of conditions, we would not be on the cusp of, at the very least, a small victory against Rett syndrome, though I believe it will be a much bigger victory than anticipated.

In ending, I know it’s hard to wait and it seems like things are taking for ever, but everyday we get closer to a definitive endpoint, not some “if” hanging over our heads. It’s going to happen; it’s going to be a game changer for all those who participate and for all those who have to wait for it to get to market. It’s a given, that each child will have a somewhat different response, as true with all medications, but it is my fundamental belief that all children will respond positively in some way. Hang on, we’re almost there.


Posted in Acadia, anxiety in Rett syndrome, Ataxia, boys with Rett syndrome, breakthrough therapy, Communication, cure, FDA, Fragile X, hope, margaret brimble, Neuren Pharmaceuticals, Rett Research, Rett Syndrome,,, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial, Trofinetide Phase III, Trofinitide, Uncategorized | Tagged , , , , , , , , , , , , , , , , , , , , , | Leave a comment

Sarizotan Trial #4: Almost There!!

As we head into the new year, I just wanted to give you all an update on the Sarizotan trial- STARS- for daytime apnea (breath-holding). I’m pleased to announce that Newron only needs 8 (or less) more participants to complete their trial.

About a month ago, I attended the Rett syndrome in-service day in Pomona. It was an informative day with multiple presenters on a wide variety of topics. One of my favorite moments was watching some of the younger girls participate in an eyegaze class. How far we have come since Katelin was first diagnosed!

My main reason for going, though, was to get an update on the Sarizotan trial and the coming Trofinetide trial. (more on that in a later post) I was so happy to get to sit down with Dr. Stephen Graham,
Executive Director, Clinical Development of Newron Pharmaceuticals US team. My first ever interview! Ok, maybe not a REAL interview, but he was happy to sit down with me and answer some questions that I had so I could give you guys information you might find useful.

First, let me start by saying how truly thankful I was that Dr. Graham is so committed to finding out all that he could about Rett syndrome that he would attend when he was not a presenter. I look for commitment when I talk to doctors and other professionals. For instance, it bugs me that the behavioral specialist Katelin currently has knows nothing about Rett syndrome and has not read up on it, even though he is “confused” on what approach to take with a non-verbal adult unable to even accurately and immediately respond to prompts. Needless to say, my stance is if you say you’re concerned about my child prove it. In talking with Dr. Graham on a couple of occasions, his passion is clear. Not only is he in contact with Rett but has been in touch with another premier Rett syndrome organization headed by Dr. Helen Leonard in Australia.

The first thing I found out is that I say Sarizotan wrong. I’ve tried now, many times, to say it correctly and well, I can’t (yet!) But, on to more serious things.

Since Katelin isn’t in this study, nor does she suffer from daytime apnea, I had questions about the medication itself and what I would want to know going into a trial- its safety profile being #1 on my list. Dr. Graham went into great detail about the history of Sarizotan in trials its tolerability and excellent safety profile.

I know some participants must have feeding tubes, so I wondered how it’s administered. He let me know that it is an oral capsule that can be dissolved. While it may seem obvious, he did confirm that the medication does pass through the blood/brain barrier. For those who may be unfamiliar with this term…
“The blood-brain barrier (BBB) is a protective barrier which is designed to keep the environment in the brain as stable as possible. It prevents many dangerous substances from entering the brain, protecting the brain from an assortment of potential risks…” This barrier is one of the biggest hurdles in treating brain disorders with medication.

I asked him about the guidelines or reporting criteria that they (Newron) was using to (hopefully) show efficacy. Dr. Graham explained that participants would be wearing a monitoring system, in addition to multiple “scales” ie:
*Caregiver-rated Impression of Change (CIC): 7-point scale requiring the caregiver to rate how much the patient’s illness has improved or worsened relative to the baseline state. * Motor-Behavioral Assessment Scale:I. Behavioral/Social Assessment – 16 items II. Orofacial/Respiratory Assessment – 7 items III. Motor Assessment/Physical Signs – 14 items. * Clinical Global Impression of Change (CGI-C): 7-point scale requiring the clinician to rate how much the patient’s illness has improved or worsened relative to the baseline state. * Rett Syndrome Clinical Severity Scale (RCSS): Frequency and manageability of seizures, respiratory irregularities, scoliosis, ability to walk (gait apraxia), hand use, speech and sleep; yielding total and feature-specific scores.

Monitors at least respiration, cardiac info and oxygen saturation.

The use of a bio-monitoring system is a great benefit because it provides actual objective data and efficacy monitoring does not rely solely upon subjective input. I wondered, though, if it could be affected by swallowing air and he did say it could be. Each of these devices are rented for thousands of dollars; in addition Newron provides a laptop with wifi to allow parents to download information from the recording device. So, I think it’s obvious they are sparing no expense to get the correct data. In addition they are working with to reimburse travel expenses, making every effort to lower the financial impact for families participating in the trial.

In closing our chat we talked about the fact that this trial is the largest trial ever conducted in Rett syndrome. It is an international trial with multiple sites here in the States as well as India, Italy, the UK and Australia. Newron is also sponsoring a Burden of Illness study which they will make available, for free, to any company that wishes to use it as a trial endpoint in the future. Having a standardized scale in regard to the effect on caregivers would be essential in comparing that aspect of a drug’s efficacy.

I hope that in this little series on Sarizotan, anyone considering placing their child in this study has been given enough information to make an informed decision in at least contacting the company through

I would like to thank Dr. Stephen Graham for going out of his way to answer any questions I’ve had and making himself available at the Rett syndrome in-service for my inquisitive nature. A special thank you to all the parents and caregivers who have made this huge commitment. Without you and your brave children research could not proceed and we would be so far from where we are now. Here’s to a hopeful 2019!

Posted in Breathing issues in Rett syndrome, cure, hope, Newron, Rett Research, Rett Syndrome, Rett syndrome Awareness,,, sarizotan, Trail to a Texas Trial | Tagged , , , , , , , , , , , | Leave a comment

When the Parent is a Punching Bag

All children act out towards parents, at some time in life, each of your children will most likely say they are unhappy with you. It’s cute when they’re four or five and they call you a “big stupid poop head.” How can you not smile at that. Your child is learning to use their words to get out their frustration at the level of their vocabulary. Sure, by the teen years, it’s a flat out “I HATE YOU!” as they’re learning to turn into adults, but most times that’s not the truth of it; they’re unhappy with your decision, and still haven’t quite got the maturity or experience to say… “I’m really unhappy that you won’t let me go to a sleepover just because the parents aren’t going to be home.” But, what to do with the child or adult who has no language?? When all they have is their body to use? 

This is what happens: Parents, usually the primary caregiver, gets the brunt of it. For over two weeks this has been going on, and while I am fairly certain of the reason, it can’t be proven, because my child cannot speak.

So what can be done when the parent believes they know the answer, but it’s psychological? This, this is what happens.  And it’s awful, but until she can talk, I’m assured of this continuing. Because even though I hate it and it’s painful and I’m tired of the trigger and I know what’s coming, I have to endure it until she feels safe and herself again. 

And this isn’t even close to what you cannot see. The horror in her eyes as she’s doing it, the fear in her eyes that she is so out of control with fear that she must lash out. There are days I’m quite certain if she could claw my eyes out or yank all the hair off my head, she would. I know this just flies in the truth of what people see when we’re out and about, the smiles they see when she knows that what she fears is not going to happen.

I still have to go through the list, even though I can pinpoint the exact day it started, just in case… Is she hurting, how regular has she been, palpate the abdomen for things like appendicitis, constipation, watch to see if it hurts when she voids; even though I know she doesn’t have a fever. 

So, while I believe this trigger is the reason, it’s not the ONLY time something like this might happen… she may step on a lego, or be constipated, or upset that one of her teachers is gone and anger is so often the outward sign of fear, sadness, not understanding, confusion, grief, it’s not going to end until she can talk to me or a professional to find some context, a different way, a BETTER way, to show all those emotions.

It’s hard to put this out there, so many know my girl as sweet as gold, and she is. But what she also is, is afraid at times, hurting at times, confused at times. And the longer it takes me to figure it out, the worse it gets. So, I want those parents who go through this to know… you’re not alone. I know without asking that there are many parents, especially those with high-functioning children, that go through this. Reach out for ideas. We’ll be going to see a psychiatrist as soon as I can get it arranged because someone besides me needs to reassure her, she needs to hear how I feel in a neutral place. We’ve been through this before; truth be told, I want Trofinetide to hurry the fuck up for this very reason. I feel this hell she goes through can end when that becomes available, and most days I can be upbeat about it, happy with the progress, understanding that trials take time. And then there are days, I just don’t want to be beat up anymore for something I didn’t do, and my patience runs a little thin.

So, as hard as it is to write that sometimes my child attacks me, I think not to do it is a disservice to those out there who must feel very alone in this struggle. You are not alone, and you CAN reach out on the forums. Be safe, learn to protect yourself and ask for suggestions that will keep you that way. 

Posted in Acadia, anxiety in Rett syndrome, Communication, cure, Neuren Pharmaceuticals, Pain in Rett syndrome, Rett Syndrome, Rett syndrome Awareness, screaming in rett syndrome, Talking, Trail to a Texas Trial, Trofinetide, Uncategorized | Tagged , , , , , , , | 2 Comments

Sarizotan Trial #3 Let’s Get This Done!

Ok Folks!

We’re down to the wire here. This trial has taken WAAAAY too long to get done. Only about 13 participants, worldwide, left to go.

82EB9CE1-8E11-4157-ACA0-B7B2C7D62234I had the pleasure of meeting Stephen Graham, Executive Director of Clinical Development for Newron Pharmaceuticals, at the SoCal annual picnic. We had a few minutes to talk and I was impressed by his passion for this trial and his optimistic hope that it might help our children.

For those just coming on board, Sarizotan trial is targeting daytime apnea-the hyperventilation/breath-holding cycle that a lot of our children suffer from.


Here are the basic criteria:

*Body weight ≥ 10 kg
*Age ≥ 4 years
*Diagnosis of Rett syndrome based on consensus clinical criteria and patients with MECP2    duplications will not be eligible.
*Has at least 10 episodes of breathing dysrhythmia, defined by episodes ≥10 seconds of        breath holding (apnea), per hour during cardiorespiratory monitoring
*Ability to take study medication provided either as capsules or combined with                         food/drink.
*Patient is cooperative, willing to complete all aspects of the study, and capable of doing so   with assistance of a caregiver.

There are multiple sites here in the US as well as Italy, India, and single sites in the UK and Australia.

For more information on the study expectations, site information and contact info, exclusion criteria take a look a

You can also take a look at for in-depth information and a questionnaire.

If you would like to a “live” person about the trial, Emma Joyce, Senior Enrollment Specialist T (857) 496-0054 ext.637  would be more than happy to speak with you and answer all your questions.

Remember, trials don’t help if we can’t get them finished!

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Winding Down


Rett syndrome Awareness #30
October is winding down and we’ve almost made it through the month. As I sit here and write this, I know there are families who didn’t make it through, they lost their children in the days between the first of the month and now.

Winding down… there are lives out there winding down in so many ways. There are boys and girls who simply won’t make it until there’s a real treatment. There are parents on the brink of divorce because the sad fact is so many marriages of special needs parents end that way. There are parents, grandparents and other caregivers who are aging and have to wonder, sooner or later, what comes next?

Winding down… There are people out there, right now, watching their children’s skills disappear and have no idea why. They haven’t heard the words “Rett syndrome” yet. They don’t know that knowing the answer is a blessing but also a curse.

Every 90 minutes a child is born with Rett syndrome, but long before then, 9 months in fact, their clock began to tick a little bit faster than most of us. Somewhere in that instant they were made, Nature shuffled their deck of cards just a little bit wrong. But, because of research, real treatment/s are imminent. Rett syndrome’s hold on our children, the children just born and on their way, is also winding down. In this game of poker, the hand we hold has finally changed a bit in our favor.

If this were the Old West, and at the table are Rett syndrome, Rett, RSRT, Huda Zoghbi and Margaret Brimble and our kids-Rett syndrome was ahead, but it’s chips are starting to fade away. The time is coming when it’ll put all its chips on the table. I do believe there would be some sweat upon its brow. It’s only a matter of time before our kids sweep all those chips off the table. The game is winding down, I can see it. I hope you can, too.

Posted in boys with Rett syndrome, cure, fund-raising for rett syndrome, Huda Zoghbi, margaret brimble, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, Rett syndrome Awareness,,, RSRT, Trail to a Texas Trial, Uncategorized | Tagged , , , , , , , , , , , | Leave a comment

Trofinetide-WTF is UP???

So, there is a LOT of speculation, misleading and outright misrepresentation of where Trofinetide stands now. So, let’s just get through all this nonsense.

Phase III is not being held up because of Acadia.

Acadia is FUNDING Phase III in America. Yes, the trial is delayed from earlier expectations, but unexpected things come up.

At this time the age cutoff has NOT been announced.

Phase IIb (Pediatric Trial) was a success. You can read a 5 part series breaking down the results-here. 

So, to sum up all of the last five installments:
*Trofinetide improved many symptoms significantly at the 200 mg/kg dose vs placebo.
*Trofinetide is safe.
*There was no “cap” seen; meaning, improvement continued throughout the trial on the 200mg/kg dose.
*There was a correlation between how much drug was measured in the blood and improvement; lighter girls did not have as much drug in their blood despite the high dosing level. This was observed in all three Trofinetide trials and Neuren is working on a solution.
*The Natural History Study is an important data collection study and has resulted in standards that can be used in clinical trials.

Heavier girls showed more benefit:

In both trials, heavier girls got up to therapeutic levels and lighter girls did not. This issue has been corrected for Phase III with a four tiered dosing system, which I addressed with Acadia when I met with their team and Neuren’s.

There have been a TOTAL of three trials so far: Phase I: a short trial of 10 females (I think that was the number) Phase IIa (Adult) and Phase IIb (Pediatric)

The LAST Phase (Phase III) is now slated for 2H2019 (The second half of 2019)

Look, we’re all anxious. We all want this to be done “yesterday”. Children and adults are dying and we’re so close to a possible treatment. Do I want the age cutoff to be the original projected age of 45, damn straight I do, but we have to live with whatever they come out with and accept that if it’s lower there’s a very good reason/s for it. It could be that younger females showed more improvement, drastic enough that no one can doubt it; maybe they found out that older parents were not willing to subject their adult children to a trial, whatever; there will be a reason and that reason may be the very thing that gets this to all of our children, boys included, faster.

I know there are a lot of new parents out there and I also know that the parents of the pediatric trial cannot talk about the results they’ve seen in their children and there’s a good reason for that- the last phase cannot be compromised by expectation.

Now, my girl was in a “safety” trial, not efficacy. It was a short trial and yes I saw improvements. Many and drastic, but Katelin is already high functioning, so I cannot say what results will be seen by others. This whole blog almost is about Trofinetide, go back to the beginning and read and watch for yourselves if you want to know what I saw. It’s all there in video, pictures and narrative.

I BELIEVE in Trofinetide with all my heart and soul. The Pediatric Phase was phenomenal with results showing a possibility to modify the course of the disorder and no cap on improvement.

What will happen in Phase III remains to be seen. Not every girl is likely to respond the same way, but all the other phases were extremely short by any stretch of the imagination and I saw improvement within 2 days. Was Katelin for sure on the medication? I don’t know, but I sincerely doubt a placebo could make her sweat and then not make her sweat when she stopped it.

Oh, and when someone says there really wasn’t much improvement, I’d like people to think about this: Trofinetide is FIXING the neurons in the brain, regrowing the dendrites and shortening the synaptic space. How jaded someone must be if they are not awed by this. For my daughter that meant beginning to correct a 18 year chronic issue within two days. I call that phenomenal improvement. Even if I hadn’t seen improvement until the last day of her short trial I would’ve been blown away. Let’s try to keep a perspective here.







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