News

NORD Rare Disease Summit, Online Oct. 18-19, Open for Registration

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…

US Patient Groups Give Thumbs-Up to Rule Against Surprise Billing

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 Successfully Managed Patient’s Hard-to-treat Seizures

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…

Partnership Aims to Lower Out-of-Pocket Costs for Rare Disease Meds

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 Open for 2022 Eurordis Black Pearl Awards

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…

Discovery Stabilizes Sodium Channel, May Avert Seizures

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.