A Day with Acadia- Part 1: Awareness

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This is my last post of Rett syndrome Awareness month. I had hoped to do more, but Rett syndrome made sure a good amount of awareness was happening right at home, but this was going to be the last either way.

 

I like to end this month on an up note and that presented itself when I was invited to speak at Acadia in San Diego. Bill Keller, Executive Director; Patient Advocacy and Industry Relations, of Acadia wanted to do a Rett syndrome Awareness event for the employees of Acadia… because the EMPLOYEES wanted to know more about it and to get a sense of the families and children they are working so hard to help.

Now, had Bill told me the scope of the audience I still would have showed up but probably with a liiittttle bit more trepidation. And, I wouldn’t bring it up except that I want you, the reader, to understand the interest and to know that this was a voluntary event. There were so many people who wanted to be there that our interview had to be broadcast to another room, there weren’t enough chairs for everyone and people were standing AND it was being broadcast as a webinar to Princeton.

Katelin, Flynn and I were met warmly by so many of the executive staff and made to feel so very welcome. Bill had asked what kind of food Katelin liked and damn! did they get it right. My girl feasted on Chicken Alfredo and then feasted on my Chicken Alfredo, lol. and, get this… Katelin’s wheelchair was in need of some minor repair but it was making it difficult to use and Bill promptly called the maintenance dept and it was zoomed away and came back as good as new!  Thank you, Juan! There was a photographer snapping away. I’ve been sent four pictures, two of them have Flynn in them. I have to wonder if the other two are the only ones that didn’t have Flynn, lol, she was VERY taken with our boy. Needless to say, we were treated like very honored guests.

It was a big deal. Probably one of the biggest deals I’ve ever participated in. There was a briefing, a debriefing, sound checks, I almost felt like I was on a News talk show. I had a portable microphone. It was an experience I can hardly believe Katelin and I were invited to. I can only hope that I represented our community as well as it deserves.

The first thing they showed was a video of Katelin of her initial journey with Rett syndrome that I made as a NNZ-2566 trial fundraising video (I edited the very end out that was asking for donations 😉 ) Luckily, Bill had forwarded the questions to me in advance so I was prepared and I will share some of them with you.

  • Can you share with the ACADIA team some of the activities that are going on in the local community around Rett Syndrome? :

I, of course, brought up the strollathons, and golf fundraisers, the LA Feast, but other events that I feel are important for the community are events like the Paint a Purple Pumpkin, and the Paint Your Nails Purple and post a picture. These are important for our community to feel connected to each other. One of my FAVORITE events is Blue Sky Day and the message it sends- so symbolic of our climb as parents to help our children, find a cure/treatment and symbolic for our children- the effort to achieve every little advancement in their own personal lives.

I recently participated in a podcast series and shared the link with Bill- To listen to the episodes, please visit anchor.fm/remarkablepodcast or search for “Remarkable Rett” wherever you listen to your favourite podcasts.

  • What about other clinical trials and areas of research?

I mentioned GP2C here. They did a great article about the different trials going on this year. The ones specifically I mentioned were the Sarizotan trial; Anavex 2-73 we discussed the Epidiolex trial in another question. I know there are two that I missed- the Ketamine trial and Triheptanoin UX007. The Ketamine trial is something I’m excited about. I haven’t yet researched Triheptanoin UX007.

  • I know that you have been following the Rett Syndrome research space for years. What is going on that excites you about future developments?

Well, of course, I said the Trofinetide Phase III excites me, lol. But, there are other developments as well. Anavex 2-73 is an exciting possibility. One area that is intriguing is the use of stem cells and one recent practice is feeding a fresh placenta to children, you can find out more about it here- Placenta Preservation. But, what excites me the most is the sheer number of trials in some stage of development or another.

  • Can you share your opinion about a topic we hear a lot about in California which is medical marijuana? Does it have a place?

I freely admitted this is not an area that I have expertise in. Mostly of what I shared came from reading accounts of other parents. I said it definitely has a place, whether it’s Charlotte’s Web or the like or other systems of delivery, like Epidiolex. I also brought up other alternative therapies such as essential oils and other naturopathic therapies. But, because this is not an area I can really give in depth insight on, I referred to Syndi Knowlton of Utah Kids.

  • Can you share with the ACADIA team your recent introduction to Dame Margaret Brimble and her role in the development of Trofinetide?

I LOVE this story and memory. Dame Brimble is the inventor of Trofinetide and you can read more about it- A Mother Meets a Child She Will Give New Life To

  • How is Katelin doing today? What are you excited about for her future?
  • What is your greatest hope?

These two go together. First, Katelin is doing very well. She’s healthy. Her spinal rod surgery went as well as could ever be expected- her back is straight, she grew almost 6 inches and she hardly ever chokes in her sleep anymore.

My greatest hope and what I am excited about in her future are intricately linked. Trofinetide is my greatest hope. *disclaimer: her trial has not been unblinded, this is my personal opinion* I’ve seen it at work. My daughter has felt it at work. Katelin has only love and admiration for Trofinetide and the people who work on it; it’s obvious every single time we do something like this, meet people involved in the trials, when we met Margaret, when we meet James Shaw and Dr. Nancy Jones of Neuren. She KNOWS it works and she’s waiting very patiently for it. Sometimes when she’s very upset, I tell her…when Trofinetide comes, this will be alright… and she calms right down.

What am I excited about her future??? College. I fully expect that Trofinetide will enable her to communicate/speak well enough to attend classes. She is going to get to LEARN what SHE wants to learn-space, biology, maybe even writing! (be still my heart 😉 )

After our interview there was a short Q & A; the one question that struck me the most was- We’ve had several parents here now and you all seem to have such joy, why is that? Well, I did joke that we were when we were there! (In my mind I was thinking- we get a great lunch and don’t have to clean up, it’s like a vacation!) However, for me, this is why I think we are able to find joy in the face of everything we deal with- we learn that the little things are the big things and the big things that most people worry about are nothing compared to those little things. You learn that showing your child a butterfly or smelling all the different flowers while you’re out walking keeps you young and reminds you of the miracles all around you. When a butterfly lands on you it can sustain you, because really, isn’t that a small miracle? Simply put we learn to be present in the moment and find the joy.

Recently, there’s been a disconnect in our community and I want to say, this experience proves to me that our hope is dependent upon all the different facets that parents promote: fundraising, research, awareness; even if it doesn’t always look like we think it will.

Without funding there is no research or trials, without trials there is no hope, and awareness isn’t needed just for those things to occur; it’s for people like the employees of Acadia- people who are going to work every day to save our children, who want to know why they should, who deserve to be told that we are grateful; people who deserve to see the children they are going to give a voice to; people who deserve to know that we are committed right alongside them to help our children, that we are not just waiting for others to be our saviors. Neuren and Acadia, among other companies, deserve to be made aware that yes they get a paycheck, but there’s a reward of a lifetime waiting at the end of trials- saving thousands of lives. Not just, hopefully, that they survive better but that they truly get to live.

Watch for: A Day with Acadia- Part 2; Assurance! Coming next week!~

 

 

 

 

This entry was posted in Acadia, Anavex, Anavex 2-73, Communication, Dame Margaret Brimble, fund-raising for rett syndrome, gp2c, hope, ketamine, kyphosis, Neuren, Neuren Pharmaceuticals, Newron, NNZ-2566, NNZ-2566 trial, Remarkable Rett-a podcast series, Rett Research, Rett Syndrome, Rett syndrome Awareness, rettsyndrome.org, RSRT, sarizotan, service dog, Talking, Trail to a Texas Trial, Trofinetide, Trofinetide Phase III. Bookmark the permalink.

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