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Epigenetic Silencing of RFX7 Defines a Transcriptional Axis Linking Lactate Metabolism to Immune Checkpoint Therapy in Glioblastoma [PDF]

open access: yesAdvanced Science
Glioblastoma (GBM) is a highly aggressive brain tumor characterized by rapid proliferation, diffuse invasion, and robust immunosuppression. Although excessive aerobic glycolysis and lactate accumulation are known to contribute to an immunosuppressive ...
Liying Han   +20 more
doaj   +3 more sources

Efficacy of stiripentol, fenfluramine, and their combination on clinical outcomes in Dravet syndrome: A preliminary report. [PDF]

open access: yesEpilepsia Open
Abstract Objective Stiripentol and fenfluramine are approved treatments for Dravet syndrome (DS), but real‐world data comparing their effectiveness and combined use remain limited. Our study aims to explore associations between treatment with stiripentol, fenfluramine, and their combination and clinical outcomes in patients with DS.
Surdi P   +7 more
europepmc   +2 more sources

Stiripentol use in Dravet syndrome patients in the USA: Results of a real‐world study [PDF]

open access: yesEpilepsia Open
Objective Dravet syndrome (DS) is a severe developmental and epileptic encephalopathy with a high seizure burden and mortality risk. Stiripentol, one of the first DS‐specific therapies, received FDA approval in 2018 but its real‐world use and impact post‐
Elaine Wirrell   +11 more
doaj   +2 more sources

Efficacy and safety of fenfluramine in Dravet syndrome: The impact of patient clinical characteristics. [PDF]

open access: yesEpilepsia Open
This graphical abstract provides an overview of the content from this post hoc analysis evaluating the efficacy and safety of fenfluramine in patients with Dravet syndrome stratified by age, number of previously attempted antiseizure medications, and SCN1A pathogenic variant status using data pooled from the three pivotal randomized controlled trials ...
Nabbout R   +20 more
europepmc   +2 more sources

Safety of Drugs Used in Difficult-to-Treat Epileptic Syndromes: A Disproportionality Analysis Using the Eudravigilance Database [PDF]

open access: yesPharmaceuticals
Background/Objectives: Difficult-to-treat epileptic syndromes include conditions typically emerging in the first years of life and are characterized by a high rate of drug refractoriness. This study aimed to better define the safety profile of drugs used
Arianna Scala   +20 more
doaj   +2 more sources

Efficacy of Stiripentol Beyond Dravet Syndrome: A Retrospective Medical Record Review of Patients with Drug-Resistant Epilepsies [PDF]

open access: yesNeurology and Therapy
Introduction Stiripentol is approved as an add-on therapy with clobazam and/or valproate for seizures associated with Dravet syndrome (DS). Evidence of stiripentol efficacy in other drug-resistant epilepsies is limited.
Víctor Soto-Insuga   +7 more
doaj   +2 more sources

Employing zebrafish to understand genetic drivers of epilepsy-related comorbid behaviors [PDF]

open access: yesFrontiers in Pharmacology
Children with epilepsy frequently experience a range of significant comorbidities beyond seizures, such as motor dysfunction, cognitive impairment, and neurodevelopmental delays. In some cases, these comorbidities contribute more significantly to overall
Chinwendu Ononuju   +5 more
doaj   +2 more sources

From first seizure to specific antiseizure medication in Dravet syndrome: Quantifying delays in the DS'coverED study. [PDF]

open access: yesEpilepsia Open
Abstract Objective Dravet syndrome (DS) is a rare early‐onset developmental and epileptic encephalopathy with persistent delays between seizure onset and diagnosis. The DS'coverED study aimed to characterize current diagnostic timelines by examining each step and its duration, identifying residual barriers, and actionable solutions to optimize the ...
Christodoulou L   +9 more
europepmc   +2 more sources

Practical consensus recommendations for polytherapy involving stiripentol in Dravet syndrome: A nominal group approach [PDF]

open access: yesEpilepsia Open
Objectives Dravet syndrome (DS) is a drug‐resistant developmental and epileptic encephalopathy, often requiring three or more antiseizure medications (ASMs).
J. Helen Cross   +4 more
doaj   +2 more sources

Hypoxia-Induced Histone Lactylation Drives Cisplatin Resistance in Bladder Cancer by Promoting RBM15-Dependent m6A Methylation of IGFBP3 [PDF]

open access: yesResearch
Histone lactylation modification and RNA m6A modification play important roles in cisplatin resistance of bladder cancer (BCa). Hypoxia drives cisplatin resistance in BCa by analyzing the TCGA-BLCA cohort, where hypoxia signatures predicted poor overall ...
Jiazhu Sun   +13 more
doaj   +2 more sources

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