High-resolution nerve ultrasound revealed nerve enlargement, a sign of nerve changes, in the arms and legs of people with…
Articles by Andrea Lobo
The first patient has been dosed in a small study testing SGT-212, a gene therapy candidate being developed by…
Skyclarys (omaveloxolone) was well tolerated and associated with stable disease measures and reduced inflammation markers in adults with…
Skyclarys (omaveloxolone) may improve the function of peripheral sensory nerves — those found outside the brain and spinal cord, which…
A newly awarded $300,000 research grant will fund a study on heart fibrosis, or the accumulation of scar tissue, in…
The volume of gray matter in the brain region known as the cerebellum helps predict motor and cognitive benefits for…
The approved Friedreich’s ataxia (FA) drug Skyclarys (omaveloxolone) was found to be generally safe and well tolerated in people…
A study of heart health with vatiquinone, an investigational oral Friedreich’s ataxia (FA) therapy, found no increased risk…
The U.S. Food and Drug Administration (FDA) has given rare pediatric disease designation to PPL-001, Papillon Therapeutics’ investigational…
People with Friedreich’s ataxia (FA) may have elevated blood levels of cardiac troponin I and T, markers of heart…