Results 141 to 150 of about 127,159 (238)
ABSTRACT Savolitinib is an oral, potent, selective CNS‐penetrant MET‐tyrosine kinase inhibitor that in vitro studies indicate is metabolized by cytochrome P450 (CYP) enzymes, including CYP1A2, and by non‐CYP enzymes, such as aldehyde oxidase (AO). Savolitinib primary metabolites, M2 and M3, are formed mainly by CYP1A2 and AO, respectively. This Phase 1,
Kowser Miah +7 more
wiley +1 more source
T.P.2.09 Induced exon skipping in normal and mdx muscle
Induced exon skipping to remove or by-pass protein truncating mutations in the dystrophin gene is emerging as a potential therapy for many cases of Duchenne muscular dystrophy. It has been proposed that the compromised sarcolemma of the dystrophic muscle
Meloni, P. +4 more
core
Splicing Factor Mutations in Clonal Hematopoiesis and Progression to Myeloid Neoplasms
Splicing factor (SF)‐mutant CH is characterized by relatively late emergence and accelerated clonal expansion in older individuals. This review summarizes its clonal dynamics, disease‐associated consequences of SF mutations in myeloid neoplasms, and candidate mechanisms that may contribute to age‐dependent selection.
Yang Lyu, Yutong Zhang, Ayana Kon
wiley +1 more source
Timing matters: Exon skipping therapy is most effective when initiated early in a mouse model of Duchenne muscular dystrophy. [PDF]
Stenler S +8 more
europepmc +2 more sources
This article reports on the clinical and imaging features of tepotinib‐induced interstitial lung disease (ILD), which were evaluated by an ILD adjudication committee composed of external respiratory and radiology experts, and the committee expertly identified 35 patients with tepotinib‐induced ILD from spontaneous adverse event reports accumulated in ...
Terufumi Kato +5 more
wiley +1 more source
Pediatric Mediastinal Gray Zone Lymphoma With Germline TET2 Heterozygous Variant
Pediatric Blood &Cancer, Volume 73, Issue 11, November 2026.
Matthew R. Schuelke +9 more
wiley +1 more source
mRNA Therapeutics for Skin Rejuvenation: From Aging Atlases to Clinical Translation
ABSTRACT Skin aging is a multifactorial process characterized by the progressive decline of tissue structure, function and regenerative capacity. Although numerous interventions have been developed to improve the clinical manifestations of aging, most primarily alleviate downstream consequences rather than directly targeting the molecular mechanisms ...
Fabiana Boncimino +3 more
wiley +1 more source
Exon Skipping of Cathepsin B [PDF]
Kathrin Müntener +4 more
openaire +1 more source
Exon-skipping therapies for DMD in Kazakhstan: Progress and challenges. [PDF]
Jaxybayeva A +4 more
europepmc +1 more source

