News

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)…

CrCEST Imaging Tool May Detect Mitochondrial Function in Muscles

Creatine chemical exchange saturation transfer or CrCEST, a metabolic imaging tool, may be a valuable technique for detecting mitochondrial function across muscles in people with Friedreich’s ataxia (FA), a new study found. To date, CrCEST has not been used to assess mitochondrial capacity in people with FA. “CrCEST MRI…

Young Rapper Hopes to Inspire Others With His New Book

Jacob Thompson began to hear an incessant ticking noise emanating from the engine of his 2002 Jeep Grand Cherokee. He played music to drown it. In doing so, he ignored a looming problem that ultimately put his car out of commission. That’s the metaphor Thompson uses when he describes his…

New Deadlines Announced for FARA Grants Program

The Friedreich’s Ataxia Research Alliance (FARA) has announced upcoming deadlines for its grants program — with awards of up to $250,000 on offer — for scientists seeking to advance research into the progressive disorder that primarily affects nerves and muscles. FARA’s research program supports basic, translational, and clinical…

Researchers Make New iPSC Models of Friedreich’s Ataxia

A team of scientists at Koc University in Turkey has created three new cell models of Friedreich’s ataxia (FA) derived from patients with repeat expansions in their FXN gene. The models “will facilitate studies to understand molecular mechanisms related to FRDA [Friedreich’s ataxia] pathology [disease processes] as well as therapeutic…

FDA Grants Fast Track Status to Oral Therapy Candidate Omaveloxolone

The U.S. Food and Drug Administration (FDA) has granted its fast track designation to Reata Pharmaceuticals’ oral candidate omaveloxolone for Friedreich’s ataxia (FA). Fast track status is designed to accelerate the development of investigational therapies that address unmet medical needs in serious or life-threatening conditions. It makes Reata eligible…