Noonan syndrome 5

MONDO:0012690

Any 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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Where it sits in the disease tree

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↑ Noonan syndrome (25)

Including sub-types (15) Tagged with Noonan syndrome 5 (1)

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New pill may replace chemo for kids with brain tumors

Disease control Ongoing

This 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

Noonan syndrome 5

Where it sits in the disease tree

New pill may replace chemo for kids with brain tumors