News

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…

Truncated forms of frataxin, the protein that is lost in people with Friedreich’s ataxia (FA), were found predominantly in mouse tissues, contrasting with the dominance of non-truncated frataxin in human tissue, a study found. The findings have implications for mouse models used to mimic the condition in disease research…

The U.S. Food and Drug Administration has put a hold on the clinical development of CTI-1601, an investigational treatment for Friedreich’s ataxia (FA) that is being developed by Larimar Therapeutics. According to a press release from Larimar, the hold was put in place after Larimar notified the…

The U.S. Food and Drug Administration (FDA) has asked Reata Pharmaceuticals to request a preliminary meeting to discuss submitting a new drug application (NDA) for omaveloxolone, an investigational therapy for Friedreich’s ataxia (FA). Reata was due to hold a Type C meeting (unrelated…

Daily dosing of CTI-1601, a lab-made, modified version of frataxin — the protein lacking in people with Friedreich’s ataxia (FA) — for 13 days led to a dose-dependent increase in frataxin levels compared to placebo, top-line data from a Phase 1 study show. Patients treated with the two…

Events are underway globally to mark Friedreich’s Ataxia Awareness Month, observed each May, to heighten awareness about the neuromuscular disease and those it affects. Awareness Day is May 15. Patients, caregivers, and advocates worldwide will flex their muscles, sport temporary tattoos, and participate in research presentations — all in the…

Healx has partnered with the Friedreich’s Ataxia Research Alliance (FARA) and Ataxia UK to combine its drug discovery expertise with the organizations’ patient and scientific insights in order to develop potential treatments for Friedreich’s ataxia. “We believe that partnership and collaboration — sharing knowledge and resources —…

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…

Stealth BioTherapeutics is planning a Phase 2a clinical trial of elamipretide (SS-31) as a treatment for people with vision loss and/or heart disease associated with Friedreich’s ataxia (FA). The company expects to use data from this study — planned to start later this year — in designing a pivotal…