About DAYBUE

Maddy, age 20, living with Rett syndrome, with her familyMaddy, age 20, living with Rett syndrome, with her family

Maddy, age 21,
living with Rett
syndrome, with
her family

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Caregivers used the Rett Syndrome Behaviour Questionnaire (RSBQ) to evaluate changes in the signs and symptoms of Rett syndrome.

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Doctors used the Clinical Global Impression-Improvement (CGI-I) scale to evaluate if Rett symptoms had improved or worsened.

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The effectiveness and safety of DAYBUE were evaluated in the 12-week LAVENDER clinical trial of 187 female patients aged 5 to 20 with Rett syndrome

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Breathing

Hand movements or stereotypies icons

Hand movements or stereotypies

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Repetitive behaviors

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Nighttime behaviors

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Vocalizations

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Facial expressions

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Eye gaze

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Mood

Symptom
areas
(RSBQ)
Quinn, 5 years oldQuinn, 5 years old
Quinn5 years old Taking DAYBUE
since 2021
Clare, 8 years oldClare, 8 years old
Clare8 years old Taking DAYBUE
since 2021
Kate, 9 years oldKate, 9 years old
Kate9 years old Taking DAYBUE
since 2021
Ellie, 15 years oldEllie, 15 years old
Ellie15 years old Taking DAYBUE
since 2020
Maddy, 20 years oldMaddy, 20 years old
Maddy21 years old Taking DAYBUE
since 2020
Hand movements or stereotypies
 More control of hands. Can hold objects longer. Not grasping her body and face as much Hands are more fluid and open, can hold a fork or cup, taps on fridge when hungry More purposeful hand use. Able to use a spoon and point at and touch her tablet Quiet, calmer hands. Can hold a cup, fork and grasp and reach for things Less aggressive hand movements. More control of hands
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Repetitive behaviors
Teeth grinding much improvedHardly grinds her teeth anymore
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Breathing
Less frequent breath holding and less hyperventilatingFewer “blue spells” (breath holding)
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Nighttime behaviors
Less frequent night terrors allow her to sleep better Fewer night terrors and laughing spells - now family gets more sleep
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Vocalizations
 Increased nonverbal vocalizations, able to let people know when she wants something She makes a lot of chatty sounds. Says "fun, funny, and food" No words, but makes more sounds to respond
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Facial
expressions
Doesn't stick her tongue out as often
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Eye gaze
 Can hold her gaze to participate and engage, especially in the classroom Uses eye gaze more to convey feelings, needs, and wishes. Allows her to show family what she wants for a snack Able to use her eye-gaze device better to convey wishesUses her eyes to say yes or no and to make choices Makes choices more quickly with her eyes. More of an interest in using her eye-gaze device
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Mood
Her moods are more predictable and cries a lot less Less screaming and tantrums. Able to take an interest in the things around her More peaceful at home, in school, and in groups She's able to regulate her emotions a little better. She's happier than she used to be Mood is better, happier, much less crying for no apparent reason
Clare, age 8, living with Rett syndrome, with her momClare, age 8, living with Rett syndrome, with her mom
View DAYBUE stories
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Significant improvements in the signs and symptoms of Rett syndrome in patients taking DAYBUE vs placebo

Get connected with a Family Support Educator
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Diarrhea:

Diarrhea is a common side effect of DAYBUE that can sometimes be severe. Diarrhea may cause your child to lose too much water from their body (dehydration). Before starting treatment with DAYBUE, stop taking laxatives. Tell your doctor if your child has diarrhea while taking DAYBUE. Your doctor may ask you to increase the amount your child drinks or give antidiarrheal medicine as needed.

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Vomiting:

Vomiting is a common side effect of DAYBUE. Sometimes vomit can get into your child's lungs (aspiration), which could cause an infection (aspiration pneumonia). Tell your doctor if they have severe vomiting or if vomiting happens often.

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Weight loss:

DAYBUE can cause weight loss. Tell your doctor if you notice your child is losing weight at any time during treatment with DAYBUE.

Common side effects in the 12-week LAVENDER trialCommon side effects in the 12-week LAVENDER trial
Management guidance

Is DAYBUE right for your child?

The DAYBUE Overview Brochure provides information about DAYBUE clinical trial results and safety info

The DAYBUE Overview Brochure provides information about DAYBUE clinical trial results, safety facts, caregiver experiences, and helpful treatment tips.

The DAYBUE Starting Treatment Discussion Guide for caregivers, a tool you can use to talk to your child’s doctor about DAYBUE

The DAYBUE Treatment Discussion Guide is a tool you can use to talk to your child’s doctor about DAYBUE.

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