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Current neurologic treatment and emerging therapies in CDKL5 deficiency disorder

CDKL5 deficiency disorder (CDD) is associated with refractory infantile onset epilepsy, global developmental delay, and variable features that include sleep, behavioral disturbances, and movement disorders. Current treatment is primarily symptom-based and informed by experience in caring for this population.

Long-term safety and efficacy of add-on cannabidiol in patients with Lennox–Gastaut syndrome: Results of a long-term open-label extension trial

Lennox–Gastaut syndrome (LGS) is an epileptic encephalopathy that is often treatment resistant. Efficacy and safety of add-on cannabidiol (CBD) to treat seizures associated with LGS was demonstrated in two randomized controlled trials (RCTs). Patients who completed the RCTs were invited to enroll in this long-term open-label extension (OLE) trial, GWPCARE5 (NCT02224573). We present the final analysis of safety and efficacy outcomes from GWPCARE5.

Long-term safety and efficacy of add-on cannabidiol in patients with Lennox–Gastaut syndrome: Results of a long-term open-label extension trial

Lennox–Gastaut syndrome (LGS) is an epileptic encephalopathy that is often treatment resistant. Efficacy and safety of add-on cannabidiol (CBD) to treat seizures associated with LGS was demonstrated in two randomized controlled trials (RCTs). Patients who completed the RCTs were invited to enroll in this long-term open-label extension (OLE) trial, GWPCARE5 (NCT02224573). We present the final analysis of safety and efficacy outcomes from GWPCARE5.

Add-on cannabidiol significantly decreases seizures in 3 patients with SYNGAP1 developmental and epileptic encephalopathy

Authors: Mathieu Kuchenbuch, Gianluca D’Onofrio, Nicole Chemaly, Giulia Barcia, Théo Teng, Rima Nabbout Published in Epilepsia Open July 2020 Abstract Mutations in SYNGAP1 are associated with developmental delay, epilepsy, and autism spectrum disorder (ASD). Epilepsy is often drug-resistant in this syndrome with frequent drop attacks. In a prospective study of add-on cannabidiol (CBD), we identified…

Cannabidiol in conjunction with clobazam: analysis of four randomized‐controlled trials

Authors: Boudewijn Gunning, Maria Mazurkiewicz‐Beldzinska, Richard F.M. Chin, Hari Bhathal Charlotte Nortvedt, Eduardo Dunayevich, Daniel Checketts Published in Acta Neurologica Scandinavica September 2020 Abstract Objectives To assess the efficacy and safety profile of add‐on cannabidiol (CBD) in patients with Lennox‐Gastaut syndrome (LGS) and Dravet syndrome (DS) on clobazam and in the overall population of four…

A pediatric patient with autism spectrum disorder and epilepsy using cannabinoid extracts as complementary therapy: a case report

Authors: Juliana Andrea Ponton, Kim Smyth, Elias Soumbasis, Sergio Andres Llanos, Mark Lewis, Wilhelm August Meerholz & Robert Lawrence Tanguay Published in Journal of Medical Case Reports September 2020 Abstract   Background The pharmacological treatment for autism spectrum disorders is often poorly tolerated and has traditionally targeted associated conditions, with limited benefit for the core…

Randomized blinded controlled clinical trial to assess the effect of oral cannabidiol administration in addition to conventional antiepileptic treatment on seizure frequency in dogs with intractable idiopathic epilepsy.

Authors: Stephanie McGrath, Lisa R. Bartner, Sangeeta Rao, Rebecca A. Packer, Daniel L. Gustafson
Journal of the American Veterinary Medical Association, 1 June 2019

OBJECTIVE: To assess the effect of oral cannabidiol (CBD) administration in addition to conventional antiepileptic treatment on seizure frequency in dogs with idiopathic epilepsy. DESIGN: Randomized blinded controlled clinical trial. ANIMALS: 26 client-owned dogs with intracta…

Use of Cannabidiol in the Treatment of Epilepsy: Efficacy and Security in Clinical Trials.

Authors: Serena Silvestro, Santa Mammana, Eugenio Cavalli, Placido Bramanti, Emanuela Mazzon
Molecules, April 2019

Cannabidiol (CBD) is one of the cannabinoids with non-psychotropic action, extracted from Cannabis sativa. CBD is a terpenophenol and it has received a great scientific interest thanks to its medical applications. This compound showed efficacy as anti-seizure, antipsychotic, n…

Efficacy of artisanal preparations of cannabidiol for the treatment of epilepsy: Practical experiences in a tertiary medical center.

Authors: Giulia S. Porcaria, Cary Fub, Emily D. Doll, Emma G. Carter, Robert P. Carson
Epilepsy & Behavior, March 2018

Medically refractory epilepsy continues to be a challenge worldwide, and despite an increasing number of medical therapies, approximately 1 in 3 patients continues to have seizures. Cannabidiol (CBD), one of many constituents of the Cannabis sativa or marijuana plant, has rece…

Cannabidiol in patients with treatment-resistant epilepsy: an open-label interventional trial.

Authors: Orrin Devinsky, Eric Marsh, Daniel Friedman, Elizabeth Thiele, Linda Laux, et al
The Lancet Neurology, March 2016

BACKGROUND: Almost a third of patients with epilepsy have a treatment-resistant form, which is associated with severe morbidity and increased mortality. Cannabis-based treatments for epilepsy have generated much interest, but scientific data are scarce. We aimed to establish w…

Cannabidiol: pharmacology and potential therapeutic role in epilepsy and other neuropsychiatric disorders.

Authors: Orrin Devinsky, Maria Roberta Cilio, Helen Cross, Javier Fernandez‐Ruiz, et al
Epilepsia, June 2014

To present a summary of current scientific evidence about the cannabinoid, cannabidiol (CBD) with regard to its relevance to epilepsy and other selected neuropsychiatric disorders. We summarize the presentations from a conference in which invited participants reviewed relevant…

Alterations of endocannabinoids in cerebrospinal fluid of dogs with epileptic seizure disorder.

Authors: Felix K Gesell, Alexander A Zoerner, Christina Brauer, Stefan Engeli, Dimitros Tsikas, Andrea Tipold
BMC Veterinary Research, 26 December 2013

BACKGROUND: Epilepsy is one of the most common chronic neurological disorders in dogs characterized by recurrent seizures. The endocannabinoid (EC) system plays a central role in suppressing pathologic neuronal excitability and in controlling the spread of activity in an epile…