NOONAN SYNDROME
Clinical trials for NOONAN SYNDROME explained in plain language.
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New drug aims to boost growth in kids with noonan syndrome
Disease control Recruiting nowThis study tests vosoritide, a protein that stimulates bone growth, in 30 children with Noonan syndrome who are not growing well despite growth hormone treatment. The goal is to see if vosoritide can safely increase their growth rate over six months. Participants will receive one…
Matched conditions: NOONAN SYNDROME
Phase: PHASE2 • Sponsor: BioMarin Pharmaceutical • Aim: Disease control
Last updated Jun 27, 2026 12:32 UTC
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New study tests online therapy to ease stress for parents of kids with RASopathies
Symptom relief Recruiting nowThis study tests whether Acceptance and Commitment Therapy (ACT), delivered through a smartphone app, can help caregivers of children with RASopathies (like Neurofibromatosis type 1 and Noonan syndrome) cope with parenting stress. The trial is fully remote and involves 70 adult c…
Matched conditions: NOONAN SYNDROME
Phase: NA • Sponsor: National Cancer Institute (NCI) • Aim: Symptom relief
Last updated Jun 27, 2026 09:05 UTC
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New study tackles diagnostic maze for rare developmental disorders
Knowledge-focused Recruiting nowThis study looks at how to reduce the long and frustrating journey to a diagnosis for people with developmental abnormalities. Researchers will review past cases, collect new blood or skin samples, and use advanced genetic testing. The goal is to understand why some people remain…
Matched conditions: NOONAN SYNDROME
Sponsor: Centre Hospitalier Universitaire Dijon • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:00 UTC
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Scientists launch major study to unravel rare genetic conditions
Knowledge-focused Recruiting nowThis study aims to learn more about RASopathies, a group of genetic conditions that can cause developmental issues, birth defects, and increased cancer risk. Researchers will follow up to 500 people of any age who have or may have a RASopathy, along with their family members, for…
Matched conditions: NOONAN SYNDROME
Sponsor: National Cancer Institute (NCI) • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:06 UTC
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Noonan syndrome research: scientists launch sample collection to unlock disease secrets
Knowledge-focused Recruiting nowThis study aims to create a collection of blood and urine samples from 100 people with Noonan syndrome. Researchers will use these samples to study how the disease works and look for factors that predict how it will progress. The study does not test any treatment, but may help gu…
Matched conditions: NOONAN SYNDROME
Sponsor: University Hospital, Toulouse • Aim: Knowledge-focused
Last updated Jun 27, 2026 11:03 UTC
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Noonan syndrome study aims to uncover hidden heart risks
Knowledge-focused Recruiting nowThis study looks at cholesterol and blood sugar levels in 200 people with Noonan syndrome and related conditions, aged 2 to 35. Researchers want to see if these levels differ by age, gender, or genetic type. The goal is to improve long-term care by identifying who might be at hig…
Matched conditions: NOONAN SYNDROME
Sponsor: IRCCS Azienda Ospedaliero-Universitaria di Bologna • Aim: Knowledge-focused
Last updated Jun 27, 2026 11:01 UTC
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New scan techniques aim to solve rare heart disease mysteries
Knowledge-focused Recruiting nowThis study is testing advanced heart MRI scans to better diagnose and predict risks for people with rare heart muscle diseases. Researchers will scan 1000 participants to see if these new imaging methods can identify conditions like Fabry disease and cardiac amyloidosis more accu…
Matched conditions: NOONAN SYNDROME
Sponsor: Chinese Academy of Medical Sciences, Fuwai Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:13 UTC
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Massive study aims to unlock secrets of rare genetic disorders
Knowledge-focused Recruiting nowThis study is collecting blood, tissue, and medical information from up to 1,000 people with RASopathies—a group of genetic conditions that affect development and raise cancer risk. Researchers will store these samples and data in a database for future studies. The goal is to lea…
Matched conditions: NOONAN SYNDROME
Sponsor: Children's Hospital Medical Center, Cincinnati • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:05 UTC