News

FDA Clears Phase 1/2 Trial of Gene Therapy for FA Heart Disease

The U.S. Food and Drug Administration (FDA) has approved a Phase 1/2 clinical trial for LX2006, Lexeo Therapeutics’ investigational gene therapy for heart disease in people with Friedreich’s ataxia (FA). Lexeo submitted the trial for approval under an investigational new drug (IND) application based on promising preclinical data. The…

Q&A With RARE-X Disease Data Platform Founder, Nicole Boice

The nonprofit RARE-X is creating an easily-accessible, centralized data hub for all rare disease patient data that can help researchers answer questions about existing disorders, discover new ones, and work toward finding treatments. It was spun out of the work that Nicole Boice, founder and chief engagement officer of…

Rare Disease Day Events Bring Awareness, Equity to Patients

Since 2008, Rare Disease Day — the last day of February — has brought together patients, caregivers, family members, friends, and advocates from around the world to raise awareness and improve equity for the more than 7,000 known rare diseases that affect more than 300 million people. In 2022, the…

Reata Begins Rolling Submission of Omaveloxolone for FA

Reata Pharmaceuticals has initiated a rolling submission of a new drug application seeking U.S. approval of its experimental oral therapy omaveloxolone for people with Friedreich’s ataxia (FA). In a rolling submission, companies can submit individual sections of the application for review by the U.S. Food and Drug Administration (FDA)…

Researcher Wins $250,000 FARA Grant to Study Proprioception Loss

A researcher in Australia has received $250,000 from the Friedreich’s Ataxia Research Alliance (FARA) to use stem cells to understand the mechanisms behind the progressive loss of proprioception — the perception of the body’s position and movement — in people with Friedreich’s ataxia (FA). The two-year grant was…