ANGELMAN SYNDROME
Clinical trials for ANGELMAN SYNDROME explained in plain language.
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Gene therapy trial aims to help people with angelman syndrome communicate better
Disease control OngoingThis early-phase trial tests a gene therapy called MVX-220 in 12 children and adults with Angelman syndrome. The therapy delivers a working copy of the UBE3A gene via a single injection into the fluid around the brain. Researchers are primarily checking safety, but also looking f…
Matched conditions: ANGELMAN SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: MavriX Bio, LLC • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC
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New drug aims to control angelman syndrome long-term
Disease control ENROLLING_BY_INVITATIONThis phase 3 trial tests the long-term safety of GTX-102 (apazunersen) in 255 people with Angelman syndrome, a genetic disorder causing developmental delays and seizures. Participants have already completed an earlier GTX-102 study and will continue receiving the drug. The main g…
Matched conditions: ANGELMAN SYNDROME
Phase: PHASE3 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Jun 27, 2026 12:29 UTC
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New drug hopes to boost brain function in kids with rare genetic disorder
Disease control OngoingThis Phase 3 trial tests a drug called GTX-102 (apazunersen) in 129 children with Angelman syndrome, a genetic condition causing severe developmental delays. The study compares the drug to a sham procedure to see if it improves cognitive function and other symptoms over about 11 …
Matched conditions: ANGELMAN SYNDROME
Phase: PHASE3 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Jun 27, 2026 12:29 UTC
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Newborn screening study aims to catch rare diseases at birth
Diagnosis OngoingThis study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…
Matched conditions: ANGELMAN SYNDROME
Sponsor: RTI International • Aim: Diagnosis
Last updated Jul 02, 2026 01:00 UTC