Targeting exercise, energy, or both in Friedreich's ataxia

Schulz J, Reetz K., Targeting exercise, energy, or both in Friedreich's ataxia,  The Lancet Neurology, 25, 435-437, doi:10.1016/S1474-4422(26)00129-8

Friedreich's ataxia is a progressive, multisystem disorder causing neurological disability along with musculoskeletal, metabolic, and cardiac involvement.1 A consistent feature is markedly reduced cardiopulmonary fitness, which matters clinically because peak aerobic capacity correlates with functional limitations and might affect long-term health risks. To address impaired cardiopulmonary function, a phase-2, single-site, randomised, 2 × 2 factorial trial tested whether an individualised, remotely supervised, home exercise programme, the NAD+ precursor nicotinamide riboside, or a combination thereof could improve cardiopulmonary fitness over 12 weeks in children and adults with Friedreich's ataxia.

Safety and efficacy of individualised exercise and NAD+ precursor supplementation in patients with Friedreich's ataxia in the USA: a single-centre, 2 × 2 factorial, randomised controlled trial

Lin K, Bucha A, McSweeney K et al. Safety and efficacy of individualised exercise and NAD+ precursor supplementation in patients with Friedreich's ataxia in the USA: a single-centre, 2 × 2 factorial, randomised controlled trial The Lancet Neurology, 25, 469-481. doi:10.1016/S1474-4422(26)00082-7 

 Friedreich's ataxia is a rare, chronic, progressive, neurodegenerative condition affecting multiple organ systems, including neurological, musculoskeletal, cardiac, and endocrine systems, and is marked by low cardiopulmonary fitness. We tested the effect of exercise and NAD+ precursor supplementation with nicotinamide riboside, which have each shown benefits in animal and early clinical studies, on cardiopulmonary fitness in individuals with Friedreich's ataxia.