News

Over about two weeks, more than 74,000 people worldwide have signed a petition from the Friedreich’s Ataxia Research Alliance (FARA) requesting that omaveloxolone (omav) be made available to people with Friedreich’s ataxia (FA) as quickly as possible. The petition specifically asks Reata Pharmaceuticals to submit a new drug…

Leriglitazone, an oral treatment candidate for Friedreich’s ataxia (FA), can increase frataxin levels and reduce neuronal degeneration in cell and animal models of the disease, a study found. Along with promising clinical findings from a Phase 2 clinical trial (NCT03917225), the results support the use of leriglitazone, and the…

Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…

Lexeo Therapeutics recently launched with an initial funding of $85 million to develop gene therapies for Friedreich’s ataxia (FA) and other disorders. The proceeds will support the clinical development of three gene therapy programs, including an anticipated Phase 1 trial this year to test LX2006 for FA-associated heart…

While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State Report Card” argues that many concerns — notably out-of-pocket costs for prescription medicines and access to affordable comprehensive care — still need attention. Those were the findings of the…

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…

The Friedreich’s Ataxia Center of Excellence of the Children’s Hospital of Philadelphia (CHOP) has been awarded a total of $1.275 million to advance research and clinical care of people with Friedreich’s ataxia (FA). The grant came from the Friedreich’s Ataxia Research Alliance (FARA), the Hamilton and Finneran families, and…

Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. To help meet that need, the National Organization for Rare Disorders (NORD)’s Rare Caregiver Respite Program may be a helpful resource. The program seeks to give a…

A gene therapy that delivers a healthy version of the FXN gene can correct heart problems in a new mouse model with cardiac-specific symptoms of Friedreich’s ataxia (FA), a study reports. This animal model more accurately mirrors the early cardiac abnormalities seen in FA…

Exicure recently discussed preclinical research data and the next steps in developing XCUR-FXN, its investigational antisense oligonucleotide (ASO) therapy for Friedreich’s ataxia (FA), in a live webcast. After initiating investigational new drug (IND)-enabling studies of XCUR-FXN in late 2020 — intended to support regulatory clearance of clinical…