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A Phase 2a trial assessing Stealth BioTherapeutics‘ lead investigational product elamipretide in people with Friedreich’s ataxia (FA) is expected to initiate by year’s end. After feedback from the U.S. Food and Drug Administration, the company amended the trial’s design. It now aims to recruit 16 FA patients with…
The Rare Disease Diversity Coalition (RDDC) awarded $600,000 in grants to ease the disparities faced by rare disease patients of color. These Impact Rare Disease Solution grants will go five RDDC steering committee working groups, which aim to identify problems for rare disease communities and advocate for solutions. The five…
A crowdfunding campaign aims to raise $45,000 to support “Rare,” a documentary film featuring the struggles and achievements of people living with rare diseases and their families. Sweis Entertainment and Digital Cave Media launched the campaign — allowing filmmakers to finish producing and to release the documentary — on Kickstarter.
Reata Pharmaceuticals is planning to ask the U.S. Food and Drug Administration (FDA) to approve omaveloxolone to treat people with Friedreich’s ataxia (FA) early in 2022, following the completion of a preliminary meeting with the agency. “Omaveloxolone could be the first drug approved for the treatment of Friedreich’s…
A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and promoting the standardization of new data collection. Launched during a virtual workshop in September, the U.S. Food and Drug…
The third United Against Ataxia Hill Day will take place virtually Sept. 29, an event set aside to heighten awareness and support legislation that affects the ataxia community, including those with Friedreich’s ataxia (FA). All U.S. residents are invited to participate in the day-long advocacy event, which is a…
Football and science seem to be disparate fields of play at first glance, but the nonprofit Uplifting Athletes is finding common ground by leveraging the popularity of college gridiron games to fund research for rare diseases. Its nearly two dozen chapters — representing college football teams across the nation…
Supporters globally are gearing up for International Ataxia Awareness Day, observed annually on Sept. 25 to raise the profile of a group of progressive neurological disorders that includes Friedreich’s ataxia (FA). Patients, caregivers, and supporters will share stories on social media, advocate on behalf of the ataxia community,…
A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest therapeutic needs, but one that is often left behind. Named the Institute for Life Changing Medicines, the project was…
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…
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