News

‘Day Undefined’ Helps Search for Adaptive Products

Liam Dougherty, who has Friedreich’s ataxia (FA), and Katelyn Leader, whose sister has an undiagnosed form of spinocerebellar ataxia, are creating a new website to connect people with all types of disabilities to consumer products that can help them in their daily lives. Day Undefined co-founders Dougherty,…

Reata Granted Pre-NDA Meeting for Omaveloxolone

Reata Pharmaceuticals has been granted a preliminary meeting with the U.S. Food and Drug Administration (FDA) to discuss the submission of a new drug application (NDA) for omaveloxolone, an investigational treatment for Friedreich’s ataxia (FA). According to a company press release, the pre-NDA meeting is scheduled…

Oxford-Harrington Partnership Targets FA as First Priority

Scientists on either side of the Atlantic have teamed up, through the new Oxford-Harrington Rare Disease Centre (OHC), to address unmet needs in rare disease research and deliver “major clinical impact” to patients. And now, the international collaboration between the University of Oxford, in England, and the Harrington Discovery…

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…

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…

Advocates Lobby US Congress During Virtual Rare Disease Week

More than 600 people participated in the 10th annual Rare Disease Week on Capitol Hill 2021, held virtually July 14–22, to advocate for the rare disease community. Hosted by the EveryLife Foundation’s Rare Disease Legislative Advocates (RDLA) program, the event brings together community members from across the U.S. to…

New System May Better Measure Walking Problems

Researchers have developed a new system that might better measure walking or gait problems in people with Friedreich’s ataxia and other nerve and muscle conditions. The study describing this method was published in the journal Sensors, as “Gauging Gait Disorders with a Method Inspired by Motor Control…

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…