ANGELMAN SYNDROME
Clinical trials for ANGELMAN SYNDROME explained in plain language.
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New hope for angelman syndrome: experimental drug ION582 enters human trials
Disease control Recruiting nowThis study tests an experimental drug called ION582 in 70 people with Angelman syndrome, a genetic condition causing developmental delays and seizures. The drug is given via spinal injection to try to improve symptoms. The main goal is to check safety and how the body processes t…
Matched conditions: ANGELMAN SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: Ionis Pharmaceuticals, Inc. • Aim: Disease control
Last updated May 17, 2026 05:45 UTC
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Gene therapy trial hopes to improve communication in angelman syndrome
Disease control Recruiting nowThis study tests a new gene therapy called MVX-220 for people with Angelman syndrome, a genetic condition that affects the nervous system. The therapy is given as a single injection into the fluid around the brain and spinal cord. The trial will include 12 children (ages 4-8) and…
Matched conditions: ANGELMAN SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: MavriX Bio, LLC • Aim: Disease control
Last updated May 17, 2026 05:33 UTC
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New hope for angelman syndrome: experimental drug enters mid-stage trial
Disease control Recruiting nowThis study tests an experimental drug called GTX-102 in 60 children and adults with Angelman syndrome, a rare genetic disorder that causes severe developmental delays. The goal is to see if the drug is safe and can improve thinking, communication, and movement. Participants will …
Matched conditions: ANGELMAN SYNDROME
Phase: PHASE2 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated May 13, 2026 16:02 UTC
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Hope for angelman syndrome: major trial tests drug to unlock speech and learning
Disease control Recruiting nowThis study tests a new medicine called ION582 in 158 children and adults with Angelman syndrome, a genetic disorder causing severe developmental delays. The goal is to see if the drug can improve communication and thinking skills. Participants will receive the drug or a placebo, …
Matched conditions: ANGELMAN SYNDROME
Phase: PHASE3 • Sponsor: Ionis Pharmaceuticals, Inc. • Aim: Disease control
Last updated May 13, 2026 15:59 UTC
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Virtual coaching may ease disruptive behaviors in kids with rare genetic disorders
Symptom relief Recruiting nowThis study tests a telehealth therapy for children aged 2-12 with genetic conditions like Fragile X or Angelman syndrome who have intellectual disabilities and challenging behaviors. Over 3-6 months, parents work with a therapist via video calls to learn strategies that promote p…
Matched conditions: ANGELMAN SYNDROME
Phase: NA • Sponsor: Rush University Medical Center • Aim: Symptom relief
Last updated Apr 30, 2026 15:50 UTC
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New study tracks angelman Syndrome's yearly changes
Knowledge-focused Recruiting nowThis study follows 40 children and adults with Angelman syndrome for one year to see how their communication, motor skills, and behaviors change. Participants visit the study site every three months for tests and questionnaires, plus have brain activity measured with EEG and wear…
Matched conditions: ANGELMAN SYNDROME
Sponsor: Massachusetts General Hospital • Aim: Knowledge-focused
Last updated May 17, 2026 05:47 UTC
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Eye tracking could unlock new biomarkers for angelman syndrome
Knowledge-focused Recruiting nowThis study aims to see if eye tracking can detect differences in how children with Angelman syndrome look at social scenes compared to healthy children. Researchers will measure where children focus their gaze to identify potential biomarkers for social perception abnormalities. …
Matched conditions: ANGELMAN SYNDROME
Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated May 17, 2026 05:45 UTC
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Scientists launch Largest-Ever study on rare imprinting disorders
Knowledge-focused Recruiting nowThis study follows 2000 children and adults with rare genetic conditions called imprinting disorders, such as Silver-Russell and Prader-Willi syndromes. Researchers aim to identify common metabolic patterns and health risks like obesity or diabetes. No new treatments are tested; …
Matched conditions: ANGELMAN SYNDROME
Sponsor: Institut National de la Santé Et de la Recherche Médicale, France • Aim: Knowledge-focused
Last updated May 14, 2026 12:05 UTC