PRADER-WILLI SYNDROME
Clinical trials for PRADER-WILLI SYNDROME explained in plain language.
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New drug under observation for rare genetic disorder
Disease control ENROLLING_BY_INVITATIONThis study follows about 200 people with Prader-Willi syndrome who are taking or starting VYKAT XR. Researchers will track side effects and how the drug affects their health over time. The goal is to gather more safety information, not to test if the drug cures the condition.
Matched conditions: PRADER-WILLI SYNDROME
Sponsor: Soleno Therapeutics, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:35 UTC
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New drug pitolisant tested for safety in rare genetic disorder
Disease control ENROLLING_BY_INVITATIONThis phase 3 trial is testing the safety of pitolisant in 150 people with Prader-Willi syndrome who have already taken the drug in a previous study. Participants will receive pitolisant tablets and be monitored for side effects. The goal is to see if pitolisant is safe for long-t…
Matched conditions: PRADER-WILLI SYNDROME
Phase: PHASE3 • Sponsor: Harmony Biosciences Management, Inc. • Aim: Disease control
Last updated Jun 27, 2026 11:02 UTC
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New drug aims to curb hunger in Prader-Willi syndrome
Disease control OngoingThis phase 2 trial tests setmelanotide (Imcivree), a daily injection, in 18 people aged 6 to 65 with Prader-Willi syndrome who have obesity. The study will check if the drug is safe and helps reduce body weight and hunger over 52 weeks.
Matched conditions: PRADER-WILLI SYNDROME
Phase: PHASE2 • Sponsor: Rhythm Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 09:04 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: PRADER-WILLI SYNDROME
Sponsor: RTI International • Aim: Diagnosis
Last updated Jul 02, 2026 02:00 UTC
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Could a gentle nerve zap curb explosive outbursts in Prader-Willi syndrome?
Symptom relief OngoingThis phase 3 study tests a device that gently stimulates the vagus nerve through the skin (tVNS) to see if it can safely reduce temper outbursts in people with Prader-Willi syndrome. About 102 participants aged 10 to 40 will use either continuous or intermittent stimulation. The …
Matched conditions: PRADER-WILLI SYNDROME
Phase: PHASE3 • Sponsor: Foundation for Prader-Willi Research • Aim: Symptom relief
Last updated Jun 27, 2026 12:04 UTC