ZX008’s mechanism of action may avoid the side effects caused by other antiepileptic treatments, preclinical research suggests. The potential treatment is also being studied in a series of pivotal clinical trials. A study based on this research, “Fenfluramine diminishes NMDA…
Treatment with topiramate (Topomax), lacosamide (Vimpat) and carbamazepine enabled a young woman with Dravet syndrome to potentially stop having seizures and eased her behavioral problems, according to a case report. The study, “Retracing…
As President Trump signed the recently passed Right to Try legislation into law in a White House ceremony, Jordan McLinn of Indianapolis tried twice to embrace him. The third time, 9-year-old Jordan finally got the hug he wanted — as well as a kiss on the forehead. The video of…
Early identification and diagnosis of seizures can have major implications for their treatment and accurate prognosis. But in most cases it can be a challenge to differentiate between epileptic seizures and abnormal non-epileptic movements early in life, a review study suggests. In the study, “Benign and…
Tilray Canada has launched its newest product, Tilray 2:100, a formulation of therapeutic cannabis oil containing a high concentration of cannabidiol (CBD), which may help treat Canadian patients with conditions like Dravet syndrome. “We are excited to offer this highly concentrated CBD product to patients in Canada. We…
Finding treatments and potential cures for rare diseases is crucial, but so is the quality of patients’ lives — a rather nebulous term that means different things to different people. “Recently, there’s been much more of a focus on Quality of Life (QoL) issues, real-world evidence and patient-reported outcomes,” said…
Stiripentol is not a cost-effective treatment for Canadian Dravet syndrome patients, and decision-makers should consider funding it, according to a recent cost analysis. The study, “Economic Evaluation of Stiripentol for Dravet Syndrome: A Cost-Utility Analysis,” was published in PharmacoEconomics. Combination therapy, or the use…
Researchers have identified two significant factors that can help predict cognitive outcomes in Dravet syndrome patients: Longer treatment with antiepileptics that block sodium channels, and being younger when the first afebrile seizure occurs. The study “Influence of contraindicated…
New insights into what causes small brain vessels to leak and promotes blood-brain barrier dysfunction in people with epilepsy were revealed in a recent mouse study. Findings were published in the study, “Matrix Metalloproteinase-Mediated Blood-Brain Barrier Dysfunction in Epilepsy,” in the Journal of Neuroscience.
The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORD’s founding at a dinner tonight in Washington, D.C. The 2018 Rare Impact Awards, to be held at the Andrew W. Mellon Auditorium, will be webcast via Facebook for those…
Get regular updates to your inbox.