ANGELMAN SYNDROME
Clinical trials for ANGELMAN SYNDROME explained in plain language.
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New drug aims to unlock speech in angelman syndrome
Disease control Recruiting nowThis Phase 3 study tests an experimental drug called ION582 in 158 children and adults with Angelman syndrome, a genetic disorder causing severe developmental delays. The drug is given as a spinal injection and aims to improve expressive communication and cognitive skills. Partic…
Matched conditions: ANGELMAN SYNDROME
Phase: PHASE3 • Sponsor: Ionis Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 14:02 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 (apazunersen) in 60 people with Angelman syndrome, a genetic disorder causing developmental delays and seizures. Participants range from 1 to 65 years old and are grouped by age and genetic type. The goal is to see if the drug …
Matched conditions: ANGELMAN SYNDROME
Phase: PHASE2 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Jun 27, 2026 14:01 UTC
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New hope for angelman syndrome: experimental drug ION582 enters human trials
Disease control Recruiting nowThis study tests a drug called ION582 for people with Angelman syndrome, a genetic condition causing developmental delays and seizures. The drug is given via a spinal injection. The main goal is to check safety and how the body processes the drug. Up to 70 participants aged 0 to …
Matched conditions: ANGELMAN SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: Ionis Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 07:57 UTC
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Can a video-based therapy tame tough behaviors in kids with rare genetic disorders?
Symptom relief Recruiting nowThis study tests a virtual behavioral therapy (Functional Behavioral Training) for children aged 2-12 with genetic syndromes like Fragile X, Angelman, or Rett syndrome who have challenging behaviors. The therapy teaches parents how to identify what triggers problem behaviors and …
Matched conditions: ANGELMAN SYNDROME
Phase: NA • Sponsor: Rush University Medical Center • Aim: Symptom relief
Last updated Jun 27, 2026 08:13 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 communication, motor skills, and behaviors change. Participants visit the study site five times for tests, brain activity monitoring (EEG), and sleep tracking. The goal is to better understand the …
Matched conditions: ANGELMAN SYNDROME
Sponsor: Massachusetts General Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:35 UTC
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Eye tracking could unlock new biomarker for angelman syndrome
Knowledge-focused Recruiting nowThis study will use eye tracking to measure how children with Angelman syndrome look at social and non-social images. Researchers hope to identify abnormalities in social perception that could serve as a biomarker for future treatment trials. The study includes 40 children with A…
Matched conditions: ANGELMAN SYNDROME
Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated Jun 27, 2026 11:01 UTC
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2000-Patient study aims to uncover hidden metabolic risks in rare genetic disorders
Knowledge-focused Recruiting nowThis observational study will follow 2000 children and adults with imprinting disorders—rare genetic conditions like Silver-Russell and Prader-Willi syndromes. Researchers aim to describe the natural history of these diseases and identify common metabolic profiles, risks for obes…
Matched conditions: ANGELMAN SYNDROME
Sponsor: Institut National de la Santé Et de la Recherche Médicale, France • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:05 UTC