Noonan syndrome 5
MONDO:0012690Any Noonan syndrome in which the cause of the disease is a mutation in the RAF1 gene.
Also known as: NS5, Noonan syndrome 5, Noonan syndrome caused by mutation in RAF1, Noonan syndrome type 5, RAF1 Noonan syndrome, RAF1 gene related Noonan syndrome
15 clinical trials for this condition and its sub-types, 1 tagged with Noonan syndrome 5 itself.
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Could a short course of steroids calm a dangerous heart rhythm?
Disease control By invitation onlyThis trial tests whether an 8-week course of the immunosuppressive drug prednisone can reduce episodes of ventricular tachycardia (a potentially fatal fast heart rhythm) and improve heart function in people with non-ischemic cardiomyopathy and confirmed heart inflammation. Partic…
Phase 4 • Sponsor: Roderick Tung • Aim: Disease control
Last updated Jul 29, 2026 00:00 UTC
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New pill may replace chemo for kids with brain tumors
Disease control OngoingThis study tests a new drug called DAY101 (tovorafenib) against standard chemotherapy for children and young adults with a type of brain tumor called low-grade glioma that has a specific gene change (RAF alteration). The goal is to see if the new drug works better at shrinking tu…
Phase 3 • Sponsor: Day One Biopharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:33 UTC
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Super MRI could spot hidden heart damage
Diagnosis OngoingThis study tests whether a new, more powerful 7 Tesla MRI can better detect scarring and swelling in the heart muscle of people with cardiomyopathy. Researchers will scan 13 adults aged 20-70 to see if the higher-resolution images improve diagnosis. The goal is to see if this adv…
Sponsor: University of Pennsylvania • Aim: Diagnosis
Last updated Jun 27, 2026 09:02 UTC
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Can a gene registry unlock the secrets of childhood heart failure?
Knowledge-focused OngoingThis study gathers health information from children under 18 who have cardiomyopathy linked to mutations in the MYBPC3 gene. Researchers aim to map the disease's natural course, identify risk factors, and measure how it affects quality of life. By reviewing past and future medica…
Sponsor: Tenaya Therapeutics • Aim: Knowledge-focused
Last updated Sep 04, 2026 00:00 UTC