Video

For those looking for more detailed information regarding the actual trial results please google search "Rett Syndrome Research Trust Daybue" The RSRT have compiled a comprehensive review of the Trofinetide trial results especially for (and at the request of) parents who are trying to navigate and make sense of all the info. It seems I can't post the actual link without my comments getting deleted.

Quite remarkable that the international Rett Syndrome Foundation would put up videos like this, and yet a year on still have made no attempt to answer any questions in the comments section.

I can’t see the video on this. Is there something I can do?

Moving video. I will support!

Imaginemos empatía, amor,bendecir y colaborar....❤❤❤❤

My daughter also got rite syndrome from Philippines

Beautiful video, Julie. Sonia is lucky to have a strong mom and supporter like you. ❤💜

My heart goes out to Sonia, her family and all the families fighting for hope for their child. Beautiful heartfelt video Julie. Love you all to pieces. ❤️❤️❤️

تحياتي من الجزائر 🇩🇿 شكرا لكل من ساهم في هذا الإنجاز ،نتمنى أن نراه قريبا في الدول الفقيرة.....

My daughter has Rett syndrome, we live in Leeds, England. Maddie’s 15 years old now. When she had her first eeg at Leeds General Infirmary hospital 12 years ago, the doctor who assisted told us did some of her neurology training alongside Professor Rett

Can I get it in jamaica

My child have Retts

Thanks for the information

On the down side...... and sadly enough those are massive.

How can I find Trofinetide & Blarcamesine in the Middle East?

KEY SUMMARY FACTS OF LAVENDERTM AND LILAC-1 TRIALS The majority of patients, 61%, taking DAYBUE were rated as “no change” on the CGI-I in the LavenderTM study. 13% of patients were rated as “much improved” on the CGI-I in the LavenderTM study. No data are provided regarding which specific symptoms get better in the LavenderTM study. 85% of patients treated with DAYBUE had diarrhea and 29% had vomiting. The prescribing information contains recommendations on managing the diarrhea. In the open label extension trial, LILAC-1, 46% of patients discontinued use of DAYBUE before the end of the study. list price of treatment per child = will be between $385,075 and $924,180 per year depending on body weight.

Is anyone please able to point me to any information regarding the various results for the Rett girls who participated in the trials? Asking as a parent of a Retts girl.

When are the next two going to air?

Muchísimas Gracias, es una gran noticia escribo desde Ecuador soy madre de una niña Rett

Presumably family members and caregivers of those participating in these trials will be watching this video. So would firstly like to reiterate a massive thank you for the families that participated in these studies. What incredibly valuable work you have done, and information you have provided, for the global rett family. Secondly would like to ask any that care to ( if you are allowed to) if you could please share your experience regarding the results of the trials/treatment, and what actual improvements you have seen for the rett girls involved. Cheers

Thank you for your information, thank you to all the families, researchers and scientists who were there. We are from Austria, following your work, waiting for new news from ACADIA every day and very much looking forward to Daybue! We celebrate with you! Thanks!❤

Hello what happens your stock?

What is the status of Tayasha trials

My aunt had this until she sadly died in 1999

I also have a child with who was diagnosed with rett syndrome I'm from Zambia 🇿🇲

I'm sorry for what you're going through. If you need to talk, I will be there

Please suggest me likely pathagenic in rett syndrome... Mile rett syndrome

Can we get update on the process outcome

My apologies. I saw this after I asked my questions today 🤦🏽‍♀️ While maintaining my sanity, I want to thank you both very much for this very informative pertinent information. I will pass this on to her new physician. Appreciate you both 💜 💜

We are so encouraged by the progression of these trials. Please keep going and we hope to one day be in a pediatric trial for gene therapy.

RETT families supporting Our RettAngels around the Globe 💜👼 # October RETT Awareness Month

I am a speech pathologist for school age children and after 20 years I was introduced to the most beautiful little girl who had the diagnosis of Rett Syndrome. I studied and went to the annual meeting for parents that had a child with Rett so I would be able to work with her. Three years later I was blessed with a granddaughter who, as Clint said, was progressing through developmental milestones until the age of 18 months. She regressed and displayed the hand wringing and other manerisims that my little student did. My son asked my opinion and I told him about the syndrome and then after many specialist doctor appointments a neurologist diagnosed my granddaughter with Rett. She is 14 years old and she is such a delight. Thank you Clint for sharing by doing the PSA because Rett is not a common diagnosis and is misunderstood.

Thanks a lot for the video, we learn a little more about Trofinetide. So bad that we French people have to wait for Neuren new partnerships.

I feel like this could have been a two page FAQ. 🥴 This video was marginally helpful, but too long. Most folks are visual learners. It was difficult to sit through the extended dialogue....and I'm a doctor. I've sat through plenty of hours worth of courses in my lifetime. The FDA sounds like the epitome of a bogged down bureaucracy. I'm sick and tired of hearing about all of these reasons why we are still waiting on a potentially life-changing pharmaceutical for our Rett kids (and Rett adults!). Sincerely, A very tired, frazzled, and out-of-patience mother to a Rett girl.

I'm disappointed in you.. The FDA is what screwed all of our children.. The chemicals they have allowed for the last 50 years in America are banned in every other country. Why does America have the most autistic mentally challenged humans? You have a lost my further support. 22 years of donating money to this foundation. I'm done;

Dr obulor more grace to your work doctor thanks so much for your medication which cured my epilepsy completely after years of suffering I have finally been cured thanks doc,,

😀🙂😊

Do you know how many people were polled roughly? And is there a report for this meeting present online somewhere? Thank you

🙌 ρɾσɱσʂɱ

2022年4月27的大会求上传更新

Mi hija tenia calculos en la vesícula y el dolor se le miraba en la cara por su expresión ojitos con ojeras y la boca se le ponía al rededor de color morado del dolor y hable con el medico que me le hiciera un ultrasonido y se le detectó la vesícula y el medico la operó con rayó lacer y salio bien gracias a Dios esta saludable

SOY DE HONDURAS TEGUCIGALPA TENGO UNA NIÑA DE 26 CON EL SÍNDROME DE RETT

My brother have 3 kids all girls two of them rett syndrome victims one is not because she's haven't feed by mother in 3months my relative have same case two of them rett syndrome victims one is not because she also haven't feed by mother .now I have doubt may be there something wrong with mother feeding I main cause of rett syndrome.plzz (confirm sry for bad English)

I can definitely relate, as my daughter has definitely made me a better person. Thank you for sharing this bit with us. #RETTy2findACure 🥰

Thank you for your video. I can so identify with all you said about Keilly. Our daughter Lisa Joy is such a gift to us for all those same reasons and more. We've kept her middle name together with her first because Joy is what characterizes her the most. One of her friends greeted me one day with, "Hi Mr Joy" because she assumed Joy was her last name. I love being called that. Best to you both and to Keilly.

Phenomenal video! 💜 Thank you for doing this.

Thank you so much for sharing this 💜

💙Hello from Pennsylvania💙 Langerhans Cell Histiocytosis. Warriors Stay strong and safe, never give up hope, never stop believing in a Cure,🌏🌍standing with you💁‍♀️🙏🏻💫Remembering all the Angels who went before us. 😔💫Thinking of all who are challanged Daily by rare diseases…you are not Alone…..🙏🏻

I’m a rett dad … god has blessed us in so many ways we may never know the full extent of the blessings. Keep on strong ! Doing great mom and dad !

I love the increase in the use of Social Media Platforms to not only get the information out there about Retts, But to also show the Human side, the people living LIFE! 😇💜💙