Brain HealthCurrent Drugs for Friedreich Ataxia Show Little Benefit After One Year
A Cochrane review pooled data from eight randomized controlled trials (seven in meta-analysis, 574 participants total) testing drugs including idebenone, omaveloxolone, CoQ10 plus vitamin E, epoetin alpha, leriglitazone, and RT001 in people with Friedreich ataxia. After 12 months, pharmacological therapies showed little to no improvement in overall ataxia scores compared to placebo (moderate-certainty evidence). Unexpectedly, upper limb dexterity showed a probable moderate improvement across three studies. Effects on heart muscle thickness, daily living activities, and exercise capacity remained highly uncertain. Adverse events causing discontinuation were rare and similar between drug and placebo groups. Evidence quality ranged from very low to moderate, meaning current treatments cannot be confidently recommended for halting disease progression.
