Novel therapeutic perspectives in Noonan syndrome and RASopathies

C Saint-Laurent, L Mazeyrie, A Yart… - European Journal of …, 2024 - Springer
Noonan syndrome belongs to the family of RASopathies, a group of multiple congenital
anomaly disorders caused by pathogenic variants in genes encoding components or …

Therapeutic efficacy of RAS inhibitor trametinib using a juvenile myelomonocytic leukemia patient-derived xenograft model

AQ Lee, H Konishi, M Ijiri, Y Li, A Panigrahi… - Pediatric Hematology …, 2024 - Taylor & Francis
Juvenile myelomonocytic leukemia (JMML) is an aggressive pediatric leukemia with few
effective treatments and poor outcomes even after stem cell transplantation, the only current …

Next-generation sequencing reveals relapse and leukemia-free survival risks in newly diagnosed acute myeloid leukemia treated with CAG regimen combined with …

S Huang, P Chen, L Wang, L Xu, N Wang… - Cancer Pathogenesis …, 2024 - mednexus.org
Background: Acute myeloid leukemia (AML) is a heterogeneous hematopoietic malignancy
whose prognosis is associated with several biomarkers. Decitabine, a deoxyribonucleic acid …

Risk-stratified Treatment for Pediatric Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia

E Stieglitz, L Pommert - The Hematologist, 2024 - ashpublications.org
STUDY DESIGN: T2020-004 is a risk-stratified, non-randomized phase I/II clinical trial
designed to examine the safety and efficacy of the MEK1/2 inhibitor trametinib in …

In vivo Targeting MEK and TNK2/SRC pathways in PTPN11 driven leukemia

BH Chang, K Thiel-Klare, JW Tyner - bioRxiv, 2024 - biorxiv.org
PTPN11 encodes for a tyrosine phosphatase implicated in the pathogenesis of hematologic
malignancies such as Juvenile Myelomonocytic Leukemia (JMML), Acute Myeloid Leukemia …