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Antiepileptic Drugs
Published in Sahab Uddin, Rashid Mamunur, Advances in Neuropharmacology, 2020
Stiripentol is used clinically as an add-on therapy for Dravet syndrome (refractory generalized tonic–clonic seizures in patients with severe myoclonic epilepsy in infancy) in patients where valproate and clobazam are unable to control symptoms satisfactorily (Plosker, 2012; Aneja and Sharma, 2013). It is still not approved by USFDA for Dravet syndrome because of many pharmacokinetic and pharmacodynamic drug interactions (Misty et al., 2018). Stiripentol also decreases the severity and frequency of tonic–clonic seizures as well as status epilepticus with a variety of epilepsy syndromes in infants and children (Goossens et al., 1999; Perez et al., 1999; Inoue et al., 2009).
Cannabis testing: Taking a closer look
Published in Betty Wedman-St. Louis, Cannabis, 2018
Scott Kuzdzal, Robert Clifford, Paul Winkler, Will Bankert
As an example, when modern medicine failed to help Charlotte Figi, a young patient who was suffering from an advanced form of epilepsy known as Dravet syndrome, Charlotte's parents found a video online about a California boy with Dravet syndrome who was being successfully treated with cannabis. At a few months old, Charlotte showed signs of the disease. At 5 years old, she could hardly walk or talk and was restricted to a wheelchair while experiencing over 300 grand mal seizures per week. After a twice-daily regimen of cannabis oil, Charlotte, now 9 years old, is not only walking—she can ride a bicycle. She is talking, feeds herself, and is down to only a few seizures per month, mostly while sleeping. This miraculous treatment was featured on CNN News in the segment, “Charlotte's Web.” Seeking similar outcomes, hundreds of families with children battling epilepsy have moved to medical marijuana legal states in search of natural cannabis cures.
Cannabis, the Plant, and Its History With Humankind
Published in Jonathan C. Beazley, Stephanie Field, Cannabis on Campus, 2018
Jonathan C. Beazley, Stephanie Field
Then, in 2011, they were contacted by the parents of Charlotte Figi, a 5-year-old girl with Dravet Syndrome, also known as myoclonic epilepsy of infancy or SMEI—a rare disorder marked by frequent, uncontrollable seizures. Charlotte would have as many as 300 seizures a week, many of them prolonged, and she had lost the ability to walk and talk and had even stopped eating. Standard medications were no longer effective, and her parents were running out of medical options. They had read online about some cases of intractable epilepsy being successfully managed in California with high-CBD/low-THC marijuana and managed to have a friend extract the oil from a small amount remaining at a dispensary in Denver. The results were dramatic; Charlotte was seizure-free for a week. But they were going to run out soon. That is when they found the Stanley brothers, who had since developed a CBD oil extract but could not yet find a market for it. This connection proved providential for both Charlotte’s family and the Stanley brothers. Word of Charlotte’s remarkable improvement spread, and, when the story was featured in a CNN television documentary, the market for the oil, now named “Charlotte’s Web,” blew up.75
SCN1A as a therapeutic target for Dravet syndrome
Published in Expert Opinion on Therapeutic Targets, 2023
There continues to be an urgent need for more effective therapies for Dravet syndrome. Although recent years have seen reports of new or re-purposed anti-seizure medications having benefit, these interventions still represent symptomatic treatments that may reduce seizure frequency but do not address the underlying molecular problem. This is crucial, as Dravet syndrome is not simply a seizure disorder, but a neurologic disease associated with many co-morbidities, including cognitive dysfunction, gait abnormalities, sleep disturbance, and early death. These issues must also be addressed in any intervention that hopes to make a truly meaningful impact on the quality of life of affected patients. Directly targeting NaV1.1 function appears to be the best path forward to a true cure for Dravet syndrome.
Stiripentol for the treatment of seizures associated with Dravet syndrome in patients 6 months and older and taking clobazam
Published in Expert Review of Neurotherapeutics, 2023
Alejandra Vasquez, Elaine C Wirrell, Paul E Youssef
Dravet Syndrome typically onsets in the first year of life and is characterized by repeated, prolonged convulsive seizures often requiring hospital and even intensive care admission. With the advances and increased availability of genetic testing, DS diagnosis is often made shortly after seizure onset which provides an opportunity to initiate early targeted therapies. STP is the only ASM with Class 1 evidence to be approved for use in patients as young as 6 months of age. In addition to reducing convulsive seizure frequency, STP also markedly reduces the number of status epilepticus episodes and associated medical complications which are more common in younger children. Thus, STP is well-positioned to be used as a first or second-line agent for most infants with DS. Furthermore, whether improved seizure control and reduced status epilepticus early in life may result in improved long-term neurodevelopmental outcomes and overall quality of life, still needs further study but is enticing!
A critical evaluation of fenfluramine hydrochloride for the treatment of Dravet syndrome
Published in Expert Review of Neurotherapeutics, 2022
An-Sofie Schoonjans, Berten Ceulemans
Currently, the therapeutic arsenal mainly consists of anticonvulsive medications and do not really address the underlying etiology. Although the underlying pathophysiology has been known for some time, only recently have the first clinical trials (phase I/II) with antisense oligonucleotides been announced. Preclinical trials showed a promising correction of the Nav1.1 haploinsuffiency in DS animal models. This is a very promising therapy and other gene therapy options will probably follow soon. Theoretically, gene therapy will prevent (or diminish) not only the convulsions, but also the cognitive decline and motor and behavioral problems. Since these therapies will be more radical (both by administration and effect) they should be started as soon as possible, it will be extremely important to get more insight into factors that predict the outcome of infants presenting with a DS. Dravet syndrome is still a spectrum and patients with a worse outcome will have a different risk-benefit ratio compared to patients in the milder spectrum. It will be more important to take this into account if the therapy becomes more invasive.