ANGELMAN SYNDROME
Clinical trials for ANGELMAN SYNDROME explained in plain language.
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New drug trial offers hope for angelman syndrome
Disease control Recruiting nowThis study is testing an experimental drug called GTX-102 in people with Angelman syndrome, a rare genetic disorder that affects development and causes seizures and movement problems. Researchers want to see if the drug is safe and if it can improve thinking, communication, behav…
Matched conditions: ANGELMAN SYNDROME
Phase: PHASE2 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Apr 01, 2026 23:11 UTC
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Brain injection gene therapy offers hope for rare neurological disorder
Disease control Recruiting nowThis early-stage trial is testing whether a single injection of experimental gene therapy (MVX-220) into the fluid around the brain can help people with Angelman syndrome. The study will enroll 12 children and adults to first check safety, then see if the treatment improves commu…
Matched conditions: ANGELMAN SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: MavriX Bio, LLC • Aim: Disease control
Last updated Mar 24, 2026 12:03 UTC
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Breakthrough drug trial offers hope for angelman syndrome families
Disease control Recruiting nowThis study is testing whether the experimental drug ION582 can improve symptoms in children and adults with Angelman syndrome, a rare genetic disorder that affects development. About 158 participants aged 2 to 50 will receive either the drug or a placebo for about 60 weeks, follo…
Matched conditions: ANGELMAN SYNDROME
Phase: PHASE3 • Sponsor: Ionis Pharmaceuticals, Inc. • Aim: Disease control
Last updated Mar 24, 2026 12:01 UTC
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Spinal injection trial offers new hope for angelman syndrome
Disease control Recruiting nowThis study is testing a new drug called ION582 for Angelman syndrome, a rare genetic disorder. The drug is given as an injection into the spinal fluid. The main goals are to see if it's safe and how the body processes it in about 70 participants, including children under 2 years …
Matched conditions: ANGELMAN SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: Ionis Pharmaceuticals, Inc. • Aim: Disease control
Last updated Mar 18, 2026 14:40 UTC
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Virtual therapy aims to ease behavioral challenges in rare genetic disorders
Symptom relief Recruiting nowThis study is testing whether a telehealth therapy program can help children with rare genetic syndromes improve their communication and reduce challenging behaviors. Children aged 2-12 with conditions like Angelman syndrome or Fragile X will work with therapists and their parent…
Matched conditions: ANGELMAN SYNDROME
Phase: NA • Sponsor: Rush University Medical Center • Aim: Symptom relief
Last updated Mar 27, 2026 12:38 UTC
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Scientists use Eye-Tracking to unlock social world of rare genetic disorder
Knowledge-focused Recruiting nowThis study aims to understand social perception in children with Angelman syndrome by tracking their eye movements while they watch videos. Researchers will compare 40 children with the syndrome to 20 healthy children to see if eye-tracking can reliably measure social attention. …
Matched conditions: ANGELMAN SYNDROME
Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated Mar 31, 2026 12:11 UTC
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Massive study launched to unlock secrets of rare genetic diseases
Knowledge-focused Recruiting nowThis French study aims to better understand a group of rare genetic conditions called imprinting disorders by tracking 2,000 patients over time. Researchers will observe patients' health patterns to identify common metabolic risks like diabetes or heart disease. The goal is to ga…
Matched conditions: ANGELMAN SYNDROME
Sponsor: Institut National de la Santé Et de la Recherche Médicale, France • Aim: Knowledge-focused
Last updated Mar 31, 2026 12:11 UTC