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.
At its first virtual investor event, biotech company Centogene set a bold mission: to cure 100 rare diseases within the next decade. A leader in the field of genetic diagnostics, Centogene used the June 22 event to present its strategic priorities, outlining its plans to speed the discovery…
In a survey of physicians and families at a rare pediatric epilepsy center, teleconsultation appointments were found to be effective and convenient, although the absence of physical exams and in-person evaluations were seen as definite drawbacks. “Despite some limitations, it is most likely that TC [teleconsultation] become a new part…
The Dravet Syndrome Foundation (DSF) is accepting applications for its Patient Assistance Grants (PAG) program that provides financial aid to people with Dravet syndrome who need medical equipment, therapy devices and educational tools. Since 2009, the PAG program runs from March 1 to Dec. 1 or until the funds…
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