The U.S. Food and Drug Administration (FDA) has provided Larimar Therapeutics with clear expectations for an application seeking accelerated…
Articles by Steve Bryson, PhD
A high dose of the Friedreich’s ataxia (FA) treatment Skyclarys (omaveloxolone), far above the approved dose to match…
Scientists have designed molecules to correct the effect of DNA expanded repeats that cause Friedreich’s ataxia (FA), according to…
The U.S. Food and Drug Administration (FDA) granted priority review to PTC Therapeutics‘ application seeking the approval of…
Skyclarys (omaveloxolone) improves heart function in Friedreich’s ataxia (FA) by reducing abnormal heartbeats and stabilizing heart rhythm, a…
Throughout 2024, Friedreich’s Ataxia News brought readers detailed coverage of the latest clinical research related to Friedreich’s ataxia (FA),…
Cardiomyopathy in people with Friedreich’s ataxia (FA) significantly associated with a shortening of chromosomal telomeres —  the “tips”…
Reduced muscle heath, physical inactivity, and more belly fat lowered sensitivity to insulin, a risk factor for diabetes, among adults…
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to PPL-001, Papillon Therapeutics‘ cell therapy for…
Widespread scar tissue in the lower left chamber of the heart — the left ventricle — correlated with genetic disease…