Gbolahan Olatunji, MD, FRSPH’s Post

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

Update on the Theranexus’ Batten-1 project in CLN3 Batten’s disease  : 1️⃣ Theranexus and Beyond Batten Disease Foundation communicated their promising results of the Phase 1/2 trial in 6 young adult CLN3 patients, at the annual meeting of the Child Neurology Society at San Diego: - a strong safety profile - a clear target engagement - suggestive of a stabilization of disease progression 2️⃣ Additional case reports from expanded use of miglustat in younger CLN3 Batten disease patients, suggesting a possible stabilization of visual acuity, and confirming the interest of visual acuity as the primary endpoint of our phase 3. 🖊 For Professor Gary Clark, the trial’s principal investigator and Chief of Child Neurology at Texas Children's Hospital : "Upon confirmation in a larger trial, these results with Batten-1 will be a breakthrough in the management of CLN3 Batten disease". 🌍 Batten-1 is the most advanced drug in development for the 2000 kids with CLN3 Batten's disease, with a potential worldwide market of 500-800 million $. 💊 Theranexus and Beyond Batten Disease Foundation are preparing their pivotal phase 3 and exploring options to have the necessary resources  to launch this study. Julien V. Marie Sebille Werner Rein Franck Mouthon Gary Clark Craig Benson Mary Beth Kiser Ineka Whiteman PhD

📃Scientific paper: Prevalence, Clinic-Etiological Spectrum and Outcome of Pediatric Metabolic Epilepsy – A Single Centre Experience Abstract: INTRODUCTION: Inborn errors of metabolism (IEM) are a rare cause of epilepsy in pediatric age group. Prompt diagnosis is essential, as some of these disorders are treatable. AIM: To determine the prevalence, clinical, and etiological profile of metabolic epilepsy in children. METHODS: A prospective observational study of children with new onset seizures diagnosed as inherited metabolic disorder in a tertiary care hospital, South India. RESULTS: Among 10,778 children with new onset seizures, 63 (0.58%) had metabolic epilepsy. The male female ratio was 1.3:1. Onset of the seizures were in neonatal period in 12 (19%), infancy in 35 (55.6%), and between one and 5 years of age in 16 (25.4%) children. Generalised seizures were seen in 46 (73%), followed by multiple seizure types (31.7%). The associated clinical features included developmental delay in 37 (58.7%), hyperactivity in 7 (11%), microcephaly in 13 (20.6%), optic atrophy in 12 (19%), sparse hair and/or seborrheic dermatitis in 10 (15.9%), movement disorder in 7 (11%), and focal deficit in 27 (42.9%) patients. Magnetic resonance imaging brain was abnormal in 44 (69.8%) and diagnostic in 28 (44.4%) patients. Causative metabolic errors included vitamin responsive errors in 20 (31.7%), disorders of complex molecules in 13 (20.6%), amino acidopathies in 12 (19%), organic acidemias in 10 (16%), disorders of energy metabolism in 6 (9.5%), and peroxisomal disorders in 2 (3.2%) patients. With specific treatment, seizur... Continued on ES/IODE ➡️ https://etcse.fr/9Rb ------- If you find this interesting, feel free to follow, comment and share. We need your help to enhance our visibility, so that our platform continues to serve you.

Recent reports suggest IV ketamine may be "promising for severe refractory headache in children." -- promising, but a word of caution at the end... According to findings reported in a meeting (April 15) at the American Academy of Neurology, "IV ketamine led to in a 50% reduction in pain at discharge, with nearly 2/3 of patients having no recurrence within 30 days." 👉 THE STUDY: The retrospective study analyzed 68 treatment sessions involving 41 patients aged 5-21 years, predominantly girls with chronic migraine without aura. Patients were administered a median dose of 0.25 mg/kg/hr of IV ketamine over a median duration of 3 days. 👉 THE FINDINGS: Approximately 50% reduction in pain scores at discharge, and two-thirds of patients experienced no headache recurrence within 30 days. While this research is promising, it's imperative to be exceedingly cautious when utilizing pharmaceuticals with and for adolescents. I firmly believe that, while these treatments can offer significant relief and represent a major advancement in pediatric care, they should ideally be considered when other integrative approaches have been explored. Prioritizing nutrition, lifestyle adjustments, and other non-pharmacological therapies can often provide foundational support for the young patient's health. It's only when these avenues have been thoroughly explored and if symptoms persist, it is then that we might consider advanced pharmaceutical interventions. Ensuring a balanced approach that prioritizes the overall well-being and long-term health of children is essential #ketamine #ketamineassistedtherapy #chronicpain #migraine #refractoryheadache

JULY is #Sarcoma Awareness Month 📣 💡 Check out this video from #ASCO2024 | Overview of managing #CNS #metastases in #pediatric #Ewingsarcoma🎥 Leonora Slatnick, MD, University of Utah provides an overview of managing #centralnervoussystem (CNS) #metastases in #pediatric patients with #Ewingsarcoma (ES) The lack of standardized management and the poor outcomes for these patients are highlighted 📉 Although outcomes may differ from other metastatic or relapsed diseases, the small cohort size makes it unclear One case involved a patient with incomplete resection who underwent high-dose #chemotherapy with #stemcell rescue and achieved long-term survival, which is unusual 🔦 Learn more 👉 https://lnkd.in/dm347SnH #VJOncology #Oncologynews #SarcomaAwarenessMonth

New Real-World Evidence Confirms AUSTEDO's Effectiveness in Huntington's Disease Treatment - https://lnkd.in/gYzeM2Eq Great read about AUSTEDO's effectiveness in treating Huntington's disease. #Teva Pharmaceuticals really hit the mark with their Phase 4 START study, presented at the 2024 AAN Annual Meeting. It's incredible to see the positive impact AUSTEDO (deutetrabenazine) Tablets are having on patients, especially with 80% reaching optimal dosing within just four weeks using the 4-week Titration Kit. #Realworldevidence backing its effectiveness, safety, and patient satisfaction is a game-changer. Kudos to Teva for leading the charge in improving treatment options for Huntington's disease! #HuntingtonsDisease #AUSTEDO #RealWorldEvidence #TevaPharmaceuticals #Neurology #Healthcare #PatientOutcomes #VMAT2Inhibitor

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

📃Scientific paper: Prevalence, Clinic-Etiological Spectrum and Outcome of Pediatric Metabolic Epilepsy – A Single Centre Experience Abstract: INTRODUCTION: Inborn errors of metabolism (IEM) are a rare cause of epilepsy in pediatric age group. Prompt diagnosis is essential, as some of these disorders are treatable. AIM: To determine the prevalence, clinical, and etiological profile of metabolic epilepsy in children. METHODS: A prospective observational study of children with new onset seizures diagnosed as inherited metabolic disorder in a tertiary care hospital, South India. RESULTS: Among 10,778 children with new onset seizures, 63 (0.58%) had metabolic epilepsy. The male female ratio was 1.3:1. Onset of the seizures were in neonatal period in 12 (19%), infancy in 35 (55.6%), and between one and 5 years of age in 16 (25.4%) children. Generalised seizures were seen in 46 (73%), followed by multiple seizure types (31.7%). The associated clinical features included developmental delay in 37 (58.7%), hyperactivity in 7 (11%), microcephaly in 13 (20.6%), optic atrophy in 12 (19%), sparse hair and/or seborrheic dermatitis in 10 (15.9%), movement disorder in 7 (11%), and focal deficit in 27 (42.9%) patients. Magnetic resonance imaging brain was abnormal in 44 (69.8%) and diagnostic in 28 (44.4%) patients. Causative metabolic errors included vitamin responsive errors in 20 (31.7%), disorders of complex molecules in 13 (20.6%), amino acidopathies in 12 (19%), organic acidemias in 10 (16%), disorders of energy metabolism in 6 (9.5%), and peroxisomal disorders in 2 (3.2%) patients. With specific treatment, seizur... Continued on ES/IODE ➡️ https://etcse.fr/9Rb ------- If you find this interesting, feel free to follow, comment and share. We need your help to enhance our visibility, so that our platform continues to serve you.

During the 76th Annual Meeting of the American Academy of Neurology (AAN) in 2024, Aquestive Therapeutics unveiled an abstract detailing the findings of a crossover study assessing the impact of seizures on the absorption of their product candidate, LIBERVANT (diazepam) buccal film, for the treatment of epilepsy in children. To know more about Pediatric Epilepsy Treatment Advancements, click: https://lnkd.in/gsU45BR2 #AAN2024 #AquestiveTherapeutics #NeurologyMeeting #Denver #EpilepsyTreatment #LIBERVANT #SeizureImpact #CrossoverStudy #BuccalFilm

📃Scientific paper: Menorrhagia as main presentation sign of severe hypothyroidism in a pediatric patient: a case report Abstract: Background The relative high frequency of menstrual irregularities in the first two–three years after menarche may lead to the risk of underestimation of associated pathological conditions, which are always to be accurately researched with careful examination and anamnesis. The association between menstrual irregularities and hypothyroidism is described in literature but the available data are scarce and mainly based on adult case series. It is described that low plasma levels of thyroid hormone can shift the hemostatic system towards a hypocoagulable and hyperfibrinolytic state and seem to lead to an increased bleeding risk. Case presentation This case report describes the case of a thirteen years old girl who presented to our Emergency Department complaining of menorrhagia for the last fifteen days, leading to severe anemia. The objective examination revealed clinical signs of hypothyroidism and a severe short stature, lower than mid-parental height, with stunting of growth and a significant bone age delay. Blood exams and thyroid ultrasound were consistent with the diagnosis of severe hypothyroidism in autoimmune thyroiditis with acquired von Willebrand syndrome, growth hormone deficiency. Magnetic resonance showed pituitary functional hyperplasia. The substitutive therapy with levothyroxine led to the resolution of heavy bleeding after five days and following normalization of coagulative parameters and pituitary hyperplasia. Conclusions Hypothyroidism usually pr... Continued on ES/IODE ➡️ https://etcse.fr/4y2K ------- If you find this interesting, feel free to follow, comment and share. We need your help to enhance our visibility, so that our platform continues to serve you.