Ultragenyx

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Profile & study

Participant context and how observations flow into your study team's view.

My Action Plan

Family seizure action plan

Reference

· Stay with your child and keep them safe from surroundings.

· Time the seizure from the first sign.

· If it lasts more than 5 minutes or repeats, follow the rescue medication steps agreed with your care team.

· Contact your study team afterward and log the event.

This summary is a caregiver reference. Always follow the personalized plan from your study team.

Participant Profile

Ethan M.

Age

7

Diagnosis

Angelman Syndrome

Enrollment

May 8, 2025

Study

GTX-102

Program

Apazunersen

Observation window

Week 8

Currently enrolled in the GTX-102 study.

Connected Sensors

Continuous observation between visits

  • Withings Sleep Analyzer

    Ethan · under-mattress sensor

    Live
  • Apple Watch

    Caregiver · wrist wearable · Battery 78%

    Live
  • Fitbit

    Available caregiver wearable

  • Garmin

    Available caregiver wearable

Health Data Sources

What flows into your study team's view

Child sleep sensor

Withings Sleep Analyzer · Ethan

Active
SleepAwakeningsConsistency

Caregiver wearable

Apple Watch · Caregiver

Active
SleepRecoveryActivity

Assessments

Completed during scheduled study visits

Reference

Baseline

Jun 12

65

Most recent

Aug 21

72

Next

Oct 16

Communication

72

+7

Motor

68

+4

Formal assessments are completed during scheduled study visits.

Evidence Data Flow

From caregiver to study team

Caregiver & sensor data

Continuous + caregiver-reported

SleepCaregiver sleepSeizuresCommunicationBehaviorMotorQoL

GTX-102 Study Team

Longitudinal observation platform

Study insights

Family understanding

Account & Study

  • Privacy & consent

  • Study documents

  • Caregiver account

GTX-102 (apazunersen) · Angelman Longitudinal Observation Study · Demo