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Dapagliflozin ameliorates Lafora disease phenotype in a zebrafish model.
Biomed Pharmacother
January 2025
IRCCS Stella Maris Foundation, Calambrone, via dei Giacinti 2, Pisa 56128, Italy.
Lafora disease (LD) is an ultra-rare and still incurable neurodegenerative condition. Although several therapeutic strategies are being explored, including gene therapy, there are currently no treatments that can alleviate the course of the disease and slow its progression. Recently, gliflozins, a series of SGLT2 transporter inhibitors approved for use in type 2 diabetes mellitus, heart failure and chronic kidney disease, have been proposed as possible repositioning drugs for the treatment of LD.
View Article and Find Full Text PDFClozapine is effective in treatment-resistant schizophrenia. However, clozapine has its own (well-) known side effects. We describe a case of a patient who developed epileptic seizures after starting clozapine.
View Article and Find Full Text PDFLafora Disease Presenting with Ataxia and DM1: A Case Study.
Acta Neurol Taiwan
December 2024
Zanjan University of Medical Sciences, Zanjan, Iran.
Here we presented a rare case of Lafora disease with neuropathy, ataxia and progression of symptoms into type one DM, GTCS and myoclonus during years. We believe that it is important to keep the diagnosis of Lafora disease in mind in every child presenting with myoclonus especially when mental and cerebellar deficits develop as well. Keywords: Drug-resistant seizure, Ataxia, Myoclonic jerky movements, Lafora.
View Article and Find Full Text PDFCost-Utility Analysis of Add-on Cannabidiol vs Usual Care Alone for the Treatment of Seizures in Patients With Treatment-Resistant Lennox-Gastaut Syndrome or Dravet Syndrome in the Netherlands.
J Health Econ Outcomes Res
December 2024
Jazz Pharmaceuticals UK Ltd., London, UK.
Article Synopsis
- Lennox-Gastaut syndrome (LGS) and Dravet syndrome (DS) are severe epilepsy types often seen in young children, and a CBD medication (Epidyolex®) has been reimbursed in the Netherlands since December 2022 to help treat their seizures.
- A cost-effectiveness analysis using a Markov model showed that using CBD along with usual care for LGS resulted in additional costs of €28,338 but provided increased quality-adjusted life-years (QALYs) of 1.318, making it cost-effective below the €80,000 threshold.
- For DS patients, CBD plus usual care showed a cost saving of €23,642 while increasing QAL
Fenfluramine treatment for Dravet syndrome: Long term real-world analysis demonstrates safety and reduced health care burden.
Epilepsia
December 2024
Department of Neuroscience and Medical Genetics, Children's Hospital Meyer IRCCS, Florence, Italy.
Objective: Fenfluramine (FFA), stiripentol (STP), and cannabidiol (CBD) are approved add-on therapies for seizures in Dravet syndrome (DS). We report on the long-term safety and health care resource utilization (HCRU) of patients with DS treated with FFA under an expanded access program (EAP).
Methods: A cohort of 124 patients received FFA for a median of 2.
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