Long-term clinical benefit of mavacamten for hypertrophic cardiomyopathy in a patient with RAF1-associated Noonan syndrome—case report
Hanna M Seghers, Bert Ectors, Maaike Alaerts, Johan Saenen, Emeline M Van Craenenbroeck · 2026
Read Long-term clinical benefit of mavacamten for hypertrophic cardiomyopathy in a patient with RAF1-associated Noonan syndrome—case report by Hanna M Seghers, Bert Ectors, Maaike Alaerts, Johan Saenen, Emeline M Van Craenenbroeck (2026) in the Tower of Babel, a free online library of public-domain works preserved in clean, searchable text. No sign-up. The full text is served as a plain-text file at a predictable URL, where text is present: https://antarctic-labs.com/tower-of-babel/pmc-13215470.txt.
Description
Five clinicians report long-term benefit from a heart drug in a rare syndrome in a case report from 2026. The patient, a twenty-nine-year-old woman, had a thickened heart muscle that blocked blood flow. Testing later showed Noonan syndrome, a genetic condition tied to a gene called RAF1. Such patients were left out of the main drug trials. The drug, mavacamten, calms the heart muscle's overactive squeezing.
She joined a trial and stayed on the drug for more than five years. The blockage eased, her symptoms improved, and chest pain and near-fainting stopped. No side effects appeared. The authors suggest the shared over-squeezing mechanism may make the drug useful beyond the usual genetic forms.
Catalog record
| CREATOR | Hanna M Seghers, Bert Ectors, Maaike Alaerts, Johan Saenen, Emeline M Van Craenenbroeck |
|---|---|
| YEAR | 2026 |
| COLLECTION | PAPERS |
| FORMAT | TXT |
| FILE SIZE | 14 KB |
| VERSION | 1.0 |
| LICENSE | CC BY 4.0 |
| RIGHTS | CC BY |
| SOURCE | Europe PMC |