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With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit. From diagnosis and clinical trial design to manufacturing, pricing strategies, and ethical concerns, gene therapy — both its high…

Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s highest-ranking health official said. “We have to think about how our financing system can protect those with serious and rare illnesses.

StrideBio and Sarepta Therapeutics announced a collaboration and license agreement to advance gene therapies for rare diseases of the central nervous and neuromuscular systems, including Dravet syndrome. Many genetic conditions are caused by mutations that impair the function of a particular gene — for instance, about three-quarters of Dravet…

Editor’s note: This story has been updated to add that Zogenix resubmitted a new drug application for Fintepla to the FDA in late September.  Treatment with Fintepla (ZX008) can provide a clinically meaningful and profound reduction in the frequency of seizures in Dravet syndrome patients, according to…

The National Institute of Health and Care Excellence (NICE), an arm of U.K.’s public health system, favors use of the oral cannabidiol solution Epidyolex as an add-on to clobazam to treat seizures associated with Dravet syndrome and Lennox Gastaut syndrome (LGS) in patients in England starting…

Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. Disorder: The Rare Disease Film Festival strives to eventually host a film about every one of the nearly 7,000 rare…

Treatment with Zogenix‘s Fintepla (ZX008) provides a sustained and meaningful reduction in convulsive seizure frequency in young children with Dravet syndrome, with similar clinical efficacy to that seen in older patient groups, according to results from a long-term open-label extension (OLE) study. Fintepla’s use also holds the potential…

Managing Dravet syndrome requires a multidisciplinary approach open to both pharmacological and non-pharmacological treatments, because of the severe nature of this disease and its poor prognosis, a review study reports. The study, “Dravet Syndrome: An Overview,” was published in Cureus. Dravet syndrome is a severe type of…

Neucyte and Trillium Therapeutics have established a license agreement to develop a new therapy for drug-resistant epilepsies, including Dravet syndrome and related disorders. The agent, whose name is still undisclosed, has shown promising results during studies carried out by the National Institute of Neurological Disorders and…

Ovid Therapeutics‘ investigational treatment soticlestat (OV935/5/TAK935) was found to progressively reduce seizure frequency — by up to 90% — in adults with rare and hard-to-treat epilepsies, including Dravet syndrome, after one year of treatment, according to preliminary data from the ENDYMION study. Topline results from the…