Caregiver Introduction

Hello there! My name is Cynthia, and I am the mother and caregiver of Joanie, a 3 and a half-year-old girl with Rett syndrome. I have some concerns about managing her health as her disease progresses. My daughter is receiving care from a wonderful team of experts! But life is not easy as she continues to grow and face different health issues associated with Rett syndrome. The changes I see now from when she was an infant makes me worry about what her life will be like as she gets older, and how much she will be able to say or do. I wish she had more treatment options.

Question #1

Cynthia, how old was your daughter when she was diagnosed?

Answer

Question #2

You mentioned she’s 3 years old now. Has she made any progress with crawling or walking, or has she developed any additional physical or behavioral symptoms?

Answer

Question #3

I see; how have you been managing her symptoms at home? Does anything you do seem to make a difference?

Answer

Question #4

But there is! A drug called trofinetide was approved in 2023 for patients 2 years and older with Rett syndrome. Have you heard about it?

Answer

Question #5

The LILAC trials found improvements in Rett symptoms based on caregiver scores with behavior questionnaires. Additionally, some caregivers provided specific feedback on observed progress made by their loved one. Would you like to hear what they reported?

Answer

Question #6

Well, improvements in eye gaze and engagement with others, along with increased hand use were reported, as well as a smaller number who learned new sounds or words. However, not everyone reported improvements.

Answer

Question #7

Yes. A larger study evaluating results from multiple trials showed that both adults and children have shown improvements in behavior and Rett syndrome severity scores. Is this treatment something you would consider for Joanie?

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Question #8

Diarrhea, vomiting, and weight loss were the most common adverse events observed in the clinical trials. Does Joanie have any issues with eating or diarrhea currently?

Answer

Question #9

Struggles with balance or coordination could lead to an inability to walk but can vary depending on disease severity. Is your child’s doctor monitoring her ability to sit, stand, and walk during her annual wellness visit?

Answer

Question #10

Yes, scoliosis is common in patients with Rett syndrome and is frequently seen in patients 16 years and older. However, it can develop at an earlier age, so it will be important to monitor for scoliosis. Do you any other questions?

Answer

This activity is provided by Med Learning Group.
This activity is supported by an education grant from Acadia Pharmaceuticals, Inc.

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