Mathieu Charvériat’s Post

Glory to God! I am delighted to share our latest article, "The Efficacy and Safety of Cannabidiol (CBD) in Pediatric Patients with Dravet Syndrome: A Narrative Review of Clinical Trials," with you all. The significance of this publication lies in its potential to enhance the treatment landscape for children with Dravet Syndrome. Dravet Syndrome, a rare and severe form of childhood epilepsy resistant to conventional treatments, has long posed a significant challenge for patients, families, and healthcare providers alike. However, our review of clinical trials shows some positive evidence that the use of Cannabidiol (CBD) holds therapeutic benefits for these patients. We reviewed the available evidence from ten clinical trials in which we observed substantial reductions in seizure frequency among pediatric patients with Dravet Syndrome treated with CBD. What's more, some patients even achieved seizure freedom, highlighting the transformative potential of this novel treatment approach. Equally important is the acceptable safety profile of CBD observed in our review, with manageable adverse events reported. This underscores the potential of CBD as a well-tolerated therapeutic option for pediatric patients with Dravet Syndrome. This publication significantly advances our understanding and management of Dravet Syndrome. It offers hope to countless families grappling with this debilitating condition and provides a foundation for further research and innovation in pediatric neurology. Kudos to the Neurology team ably led by Nicholas ADERINTO #Dravetsyndrome #Cannabinoids #pediatricneurology

Thrilled to announce the publication of our latest research article: Title: Neuro-inflammatory pathways in COVID-19-induced central nervous system injury: Implications for prevention and treatment strategies Journal:Experimental Neurology (Elsevier) Year: 2024 Metrics: Q1, Impact Factor 4.6, Cite Score 10.1 Description: This study explores the neuro-inflammatory pathways involved in COVID-19-induced central nervous system injury, highlighting potential prevention and treatment strategies.

Excited to share a recent publication in Pediatric Neurology from my time at Boston Children's Hospital: "Inaugural Patient-Reported Registry of Pediatric Opsoclonus-Myoclonus-Ataxia Syndrome: Presentation, Diagnosis, and Treatment of 194 Patients." This paper represents a novelty in rare disease research, utilizing entirely patient-reported data to yield insight into OMAS, one of several devastating pediatric #neuroinflammatory conditions our team regularly treated. One year out, I'll forever be grateful to have contributed to this important work. 📄 https://lnkd.in/ejwg9WPP #OMSLife #OMAS OMSLIFE FOUNDATION INC #opsoclonus #raredisease #neurology #psychiatry #neuroimmunology #neuroscience #iamrare

Happy to share our latest article on Lp(a) levels and ischemic stroke in children! In this case-control study, we found an association between Lp(a) and the risk of ischemic stroke in children, suggesting that high Lp(a) might be an independent risk factor for ischemic stroke. Given the large impact of ischemic stroke, we believe that the potential role of Lp(a) in pediatric stroke should be further studied, especially now that Lp(a)-lowering therapies are on the way! Many thanks to all the authors! Albert Wiegman, Robert van Gemert, Barbara Hutten, Irene Klaassen https://lnkd.in/e2QBY-am

NeuroDerm, A Mitsubishi Tanabe Pharma Group Company, left a notable mark at the 2024 American Academy of Neurology conference with groundbreaking research spotlighting the effectiveness and safety of ND0612, a 24-hour subcutaneous infusion of levodopa/carbidopa, in Parkinson's disease patients dealing with motor fluctuations. These studies offer valuable insights into ND0612's potential to enhance symptom control and overall quality of life for those with PD. In the first study (NCT04006210), the efficacy and safety of ND0612 were examined across various subgroups of Parkinson's disease patients experiencing motor fluctuations. The findings revealed a consistent positive impact of ND0612 in extending "Good On" time without troublesome dyskinesia across diverse patient demographics and baseline characteristics. This consistent treatment effect underscores ND0612's capacity to deliver meaningful clinical benefits across a broad range of Parkinson's disease patients. To know more about the breakthroughs in Parkinson's disease management, visit: https://lnkd.in/gidhYMic #Neuroderm #ParkinsonsDisease #ND0612 #MotorFluctuations #Levodopa #Carbidopa #NeurologyConference #ClinicalResearch #SymptomManagement #QualityOfLife

During the 2024 American Academy of Neurology (AAN) conference, SK Life Science, Inc. presented abstracts revealing insights into their upcoming medication, XCOPRI (cenobamate). The data showcased studies investigating the comparative bioavailability of the drug when taken as intact or crushed tablets via oral ingestion or administered through a nasogastric tube. Additionally, research evaluated cenobamate's effectiveness in pediatric patients experiencing intractable focal seizures. Furthermore, a retrospective analysis based on claims was presented, examining cenobamate's efficacy in reducing hospitalizations among individuals experiencing focal-onset seizures. To know more about the epilepsy treatment landscape, visit: https://lnkd.in/g4VWVnSE #AANconference #SKLifeScience #XCOPRI #Cenobamate #Bioavailability #OralIngestion #NasogastricTube #PediatricSeizures #IntractableSeizures #HospitalizationReduction #NeurologyResearch #FocalSeizures

Drs. Michelle Mielke and Nicole Fowler, both principal investigators for the ADACC Network, have co-authored a review for the Nature Reviews: Neurology entitled "Alzheimer disease blood biomarkers: considerations for population-level use." In this article, Drs. Mielke and Fowler discuss the utility and accuracy of blood-based biomarkers (BBMs) in clinical settings, the impact that demographic characteristics and chronic conditions like CKD, diabetes, and cardiovascular disease can have on BBM results, and the need for policies to protect patient rights and guide the future use of BBMs. Read more about their article here: https://lnkd.in/g84wjPAP

Time to treatment of the first myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD) attack may be associated with the long-term prognosis and autoimmune status of patients, according to study results published in JAMA Neurology. Read here: https://bit.ly/4hOLxD0 #MyelinOligodendrocyteGlycoproteinAntibodyAssociatedDisease #MOGAD #AutoimmuneStatus #Ophthalmology #EyeHealth #EyeCare #NeuroOphthalmology

I am pleased to share our latest publication in the European Journal of Medical Genetics: "Cardiovascular abnormalities in patients with SHANK3 pathogenic variants: Beyond neurodevelopmental disorders and epilepsy." 🫀📚 This study provides new insights into the cardiovascular risks associated with SHANK3 pathogenic variants, underlining the importance of comprehensive cardiac evaluations for these patients. A big thank you to my co-authors for their dedication and hard work! 👏 Lucy Dougherty de Miguel, Alícia Artigas Baleri, Laia Turón, Ivon Cuscó, Asun Díaz, Luísa Panadés de Oliveira and Susana Boronat. Read the full article here: https://lnkd.in/dV8ynGxw #PediatricCardiology #Genetics #Cardiomyopathy #Research #NeurodevelopmentalDisorders #Pediatrics

Identifying neuromuscular diagnoses can be difficult. The signs may be subtle variants of normal behavior and development, and may be hard to discern. Tools pediatricians can use to help recognize the signs and symptoms of #Duchenne include:   -- PPMD worked with the Centers for Disease Control and Prevention (CDC) to develop ChildMuscleWeakness.org, a tool to enable medical professionals to recognize the possible signs of neuromuscular disorders and to seek diagnosis. -- For families still in the observation stage, the American Academy of Pediatrics has developed the Physical Developmental Delays: What To Look For tool to help parents know if and when to seek additional advice and testing for their child: https://lnkd.in/ezpBE9hx -- The France Foundation has created a learning library including videos, podcasts, and infographics to demonstrate early screening, evaluation, and the ongoing role of pediatricians as a member of the patient’s care team: https://lnkd.in/eJxV5PJJ