News

Registration is now open for Global Genes‘ 2021 RARE Patient Advocacy Summit. This year’s hybrid event will be livestreamed from California Sept. 27-29, and some seats also are available for attending the event in person in San Diego. “Here you’ll have the opportunity to connect and engage with others…

Siblings of children with Dravet syndrome often grow up “in an environment permeated by stress, anxiety, and fear for their loved one’s well-being,” according to the biopharmaceutical company Zogenix. Now, a new online resource is available to help support the brothers and sisters of patients with severe rare epilepsies.

Registration is now open for the 2021 Rare Diseases and Orphan Products Breakthrough Summit, which will be held virtually Oct. 18–19. The event, also known as the National Organization for Rare Disorders (NORD) Summit, brings the rare disease community together to network and discuss developments in treatments and research…

CAMP4 Therapeutics has entered an exclusive worldwide licensing agreement to develop, manufacture, and commercialize OPKO Health’s RNA-based technology as potential disease-modifying treatments for Dravet syndrome. As part of the deal, CAMP4 has prioritized OPKO’s lead candidate for Dravet to enter clinical development. “We are delighted to enter into…

The National Organization for Rare Disorders (NORD) is applauding the Biden administration for announcing a rule to protect consumers from surprise medical billing, in a joint statement with 26 other U.S. patient organizations. The interim final rule will implement patient protections required by the No Surprises Act. Surprise…

Fintepla (fenfluramine) successfully managed the severe and difficult-to-treat seizures of a 20-year-old woman with Dravet syndrome, demonstrating the medication’s potential in real-life for treating such challenging cases, according to researchers. “The profound and sustained response to [Fintepla] seen in this patient is similar to that observed in double-blind clinical…

AllianceRx Walgreens Prime, a specialty and home delivery pharmacy, is partnering with TailorMed, a healthcare technology company, to help lower out-of-pocket prescription costs for specialty pharmacy patients. Medications attained through specialty pharmacies are those used to treat rare and chronic conditions in the U.S., and are often extremely costly. For…

Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. The 12 award categories recognize individual advocates, policy makers, researchers, organizations, and companies who work to make a difference for the global rare disease community. The deadline for nominations is Sept. 10…

Great Ormond Street Hospital (GOSH) Charity and Sparks are investing £2.5 million (about $3.5 million) in 11 medical research projects that will investigate rare childhood diseases, including Dravet syndrome. The GOSH Charity and Sparks National Call is a U.K. initiative to support medical research into pediatric rare diseases,…

Controlling a biological reaction that stabilizes part of the sodium channel in nerve cells can become a new approach to prevent epileptic seizures, according to a mouse study. This reaction, called neddylation, stabilizes the NaV1.1. subunit of the sodium channel, in which mutations are responsible for the development of Dravet syndrome.