RASopathy
MONDO:0021060Developmental syndromes caused by germline mutations (or in rare cases by somatic mosaicism) in genes that alter the Ras subfamily and mitogen-activated protein kinases that control signal transduction.
Also known as: RASopathy, Ras protein signal transduction disease, disorder of Ras protein signal transduction
94 clinical trials for this condition and its sub-types.
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Broader categories
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Experimental cancer drug study halted early
Disease control TerminatedThis early-stage study tested a new drug called PF-07284892, alone or with other medicines, in people with advanced solid tumors that had specific genetic changes. The goal was to find the safest dose and check for side effects. The study was stopped early, so results are limited…
Phase: PHASE1 • Sponsor: Pfizer • Aim: Disease control
Last updated Jun 27, 2026 13:02 UTC
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New cancer pill shows early promise, but trial halted
Disease control TerminatedThis early-stage trial tested an oral drug called TNO155, alone or with another drug (nazartinib), in 227 adults with advanced solid tumors like lung cancer, melanoma, and head/neck cancer. The main goal was to check safety and find the right dose. The study was terminated early,…
Phase: PHASE1 • Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 12:07 UTC
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Scientists dive into cells to unravel costello Syndrome's secrets
Knowledge-focused TerminatedThis study collects small skin samples from children aged 2 to 17 with Costello syndrome or a related condition. Researchers will analyze the cells to understand how a mutation in the HRAS gene affects energy use and mitochondria. The goal is to learn more about the disease's und…
Phase: NA • Sponsor: University Hospital, Bordeaux • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:05 UTC