Trametinib Therapy for Hypertrophic Cardiomyopathy and Pulmonary Hypertension in a Child With RAF1‐Related Noonan Syndrome (p.Ser257Leu): A Case Report

ABSTRACT This case describes a female infant with RAF1‐related Noonan syndrome who developed severe hypertrophic obstructive cardiomyopathy, pulmonary hypertension, and cardiorespiratory failure that responded to trametinib treatment but ultimately progressed to death following dose tapering and dis...

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Principais autores:	C. Noah Nilsson, Othman A. Aljohani, Michael A. Smith, Rachelle Durand, Inger Norlyk Sheyanth, Hythem Nawaytou, Elliot Stieglitz, Russel Valle, Sanjeev A. Datar
Formato:	Artigo
Idioma:	Inglês
Publicado:	Wiley 2026-06-01
Series:	Clinical Case Reports
Assuntos:	hypertrophic cardiomyopathy MEK inhibition Noonan syndrome pulmonary hypertension Trametinib
Acceso en liña:	https://doi.org/10.1002/ccr3.72831
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