News

Group Focuses on Rare Disease Clinical Trial Participation

Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running an endurance race they are highly motivated to complete, but these incremental burdens negatively impact their ability or willingness to…

Register Now for Global Genes’ RARE Patient Advocacy Summit

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…

New Program Supports Siblings of Rare Epilepsy Patients

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.

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…

CAMP4 Buys Global Rights to OPKO Health’s RNA-based Therapeutics

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…

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…