Download full-text PDF |
Source |
|---|---|
| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7857785 | PMC |
| http://dx.doi.org/10.1093/ckj/sfaa068 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Synthesis and LDHA Inhibitory Activity of New Stiripentol-Related Compounds of Potential Use in Primary Hyperoxaluria.
Int J Mol Sci
December 2024
Department of Inorganic and Organic Chemistry, Faculty of Experimental Sciences, University of Jaén, Campus of International Excellence in Agri-Food (ceiA3), 23071 Jaén, Spain.
Human lactate dehydrogenase A (LDHA) is a homotetrameric isozyme involved in the conversion of glyoxylate into oxalate in the cytosol of liver cells (hepatocytes) and partially responsible for the overproduction of oxalate in patients with the rare disease called primary hyperoxaluria (PH). Recently, LDHA inhibition has been validated as a safe therapeutic method to try to control the PH disease. Stiripentol (STP) is an approved drug used in the treatment of seizures associated with Dravet's syndrome (a severe form of epilepsy in infancy) which, in addition, has been drawing interest in recent years also for potentially treating PH, due to its LDHA inhibitory activity.
View Article and Find Full Text PDFA comprehensive review of evolving treatment strategies for Dravet syndrome: Insights from randomized trials, meta-analyses, real-world evidence, and emerging therapeutic approaches.
Epilepsy Behav
January 2025
Division of Child Neurology, Department of Pediatrics, University of Arkansas for Medical Sciences, Little Rock, AR, USA. Electronic address:
Dravet syndrome (DS) is a severe genetic developmental and epileptic encephalopathy, primarily caused by SCN1A gene mutations. Historically, treatments like clobazam and valproate have been used without evidence from randomized controlled trials (RCTs). However, the therapeutic landscape of DS has evolved with multiple RCTs demonstrating the efficacy and safety of three antiseizure medications (ASMs): stiripentol, cannabidiol (CBD), and fenfluramine.
View Article and Find Full Text PDFHome-based, computer-assisted cognitive rehabilitation for attention in pediatric onset multiple sclerosis: a randomized, multicenter pilot study.
Neurol Sci
February 2025
Department of NEUROFARBA, University of Florence, Largo Piero Palagi, 1, Florence, 50139, Italy.
Objective: Cognitive impairment affects approximately 30% of pediatric onset Multiple Sclerosis (POMS) patients with a negative impact on everyday life. The aim of this study was to evaluate the feasibility and effectiveness of a home-based, computer-assisted training of attention in patients with POMS.
Methods: This was a randomized double-blind study.
Use of Stiripentol in Patients with Dravet Syndrome: Common Practice Among Experts in Spain.
Neurol Ther
February 2025
Unidad de Neurología Pediátrica, Clínica Universidad de Navarra. Member of the Instituto de Investigación Sanitaria de Navarra (IdisNa), Pamplona, Spain.
Background: Despite considerable evidence for the efficacy and safety of stiripentol in Dravet syndrome (DS), some aspects of stiripentol use remain challenging in clinical practice, such as dose titration and the adjustment of concomitant antiseizure medications (ASMs) to prevent potential adverse effects.
Aim: To (1) provide practical recommendations on the initiation of stiripentol treatment in patients with DS, (2) evaluate its effectiveness in the patient, and (3) guide the management of drug interactions and other aspects of treatment monitoring.
Methods: Six Spanish neurologists (the authors) with expertise in the management of pediatric and adult patients with DS held a meeting in early 2024 to develop expert recommendations regarding the use of stiripentol in DS, based on a review of the literature and their common clinical experience.
Adverse events associated with Stiripentol in children aged 0-17 years: An analysis of a real-world pharmacovigilance database.
Epilepsy Behav
December 2024
Department of Pharmacy, The Second Affiliated Hospital of Fujian Medical University, Quanzhou, China. Electronic address:
Article Synopsis
- - The study investigates the occurrence of adverse drug events (ADEs) linked to Stiripentol (STP) in children aged 0-17 using data from several pharmacovigilance databases, finding a total of 31,990 reported ADEs.
- - Analysis reveals higher rates of ADEs related to nervous system disorders and increases significantly in children aged 3-11, with specific alarming signals like cerebellar atrophy in toddlers and changes in seizure presentation in younger children.
- - Gender differences are noted, with boys reporting more ADEs for certain medical conditions, while girls show higher incidences of skin and injury-related issues in the same age group.
Want AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!