News

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…

In addition to three recruiting clinical studies that were announced in June, an additional trial is seeking participants with Friedreich’s ataxia (FA) to investigate how the disease affects the brain, spinal cord, and cognition. Listed in a monthly newsletter released by the Friedreich’s Ataxia Research Alliance (FARA),…

The National Organization for Rare Disorders, known as NORD, was named an official charity partner of the 2021 TCS New York City Marathon, which will be held Nov. 7 both in-person and online. “Supporting charitable causes and organizations are a long-standing tradition of the TCS New York City…

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,…

The National Alliance for Caregiving, in partnership with Global Genes, has issued a free guidebook, available online, that offers resources and support for caregivers of children with rare diseases. “The Circle of Care Guidebook for Caregivers of Children With Rare and/or Serious Illnesses” was designed…

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…

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…

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…

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…

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…