ANGELMAN SYNDROME
Clinical trials for ANGELMAN SYNDROME explained in plain language.
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Gene therapy trial hopes to ease angelman syndrome symptoms
Disease control Recruiting nowThis study tests a new gene therapy called MVX-220 for people with Angelman syndrome, a genetic condition that causes severe developmental delays. The therapy is given as a single injection into the fluid around the brain. The trial will include 12 children and adults to see if i…
Matched conditions: ANGELMAN SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: MavriX Bio, LLC • Aim: Disease control
Last updated May 26, 2026 08:20 UTC
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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 18, 2026 12:08 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 aims to ease disruptive behaviors in kids with rare genetic disorders
Symptom relief Recruiting nowThis study tests a telehealth therapy program for children aged 2-12 with genetic syndromes 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 pr…
Matched conditions: ANGELMAN SYNDROME
Phase: NA • Sponsor: Rush University Medical Center • Aim: Symptom relief
Last updated May 19, 2026 11:48 UTC
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Scientists launch largest 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 describe how these conditions affect metabolism over time, including risks for obesity, diabetes, and heart …
Matched conditions: ANGELMAN SYNDROME
Sponsor: Institut National de la Santé Et de la Recherche Médicale, France • Aim: Knowledge-focused
Last updated May 26, 2026 08:34 UTC
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Eye tracking could unlock social clues in angelman syndrome
Knowledge-focused Recruiting nowThis study looks at how children with Angelman syndrome (a rare genetic disorder causing severe developmental delays) pay attention to social cues compared to healthy children. Researchers will use eye-tracking technology to see where kids look when watching social scenes. The go…
Matched conditions: ANGELMAN SYNDROME
Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated May 22, 2026 14:05 UTC
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New study tracks angelman syndrome progression in kids and adults
Knowledge-focused Recruiting nowThis study follows 40 people with Angelman syndrome over one year to see how their skills, brain activity, and sleep change. Participants visit the study site five times for tests and questionnaires, plus wear a sleep monitor at home. The goal is to better understand the natural …
Matched conditions: ANGELMAN SYNDROME
Sponsor: Massachusetts General Hospital • Aim: Knowledge-focused
Last updated May 22, 2026 14:04 UTC