Researchers at Children’s Hospital of Philadelphia (CHOP) reported encouraging results of the benefits of exercise for people with Friedreich’s ataxia (FRDA), a rare, inherited condition that gradually affects coordination, strength, and endurance, and can also involve the heart. The findings, reported recently in The Lancet Neurology, showed that a structured, home-based exercise program improved cardiorespiratory fitness over 12 weeks, with the largest gains in participants who paired training with a supplement aimed at supporting cellular energy.
FRDA disrupts the function of mitochondria – the cell’s energy “powerhouses”– and many affected individuals experience significant fatigue and poor exercise tolerance that can impact daily life. Researchers launched this study to address a long-standing gap: although individuals with FRDA are often advised to “stay active,” there has been limited data on what home-based exercise programs are feasible and safe in FRDA, especially given concerns about falls and heart strain.
Additionally, treatment options for these patients are limited. Some individuals with FRDA have experienced benefits from a supplement called nicotinamide riboside (NR), a molecule the body can convert into NAD+, a coenzyme that supports metabolism. However, little was known about its benefits when used alongside home-based exercise programs.
In the study, led by Kimberly Lin, MD, Medical Director of the Cardiomyopathy Program in CHOP’s Cardiac Center, researchers assessed 66 children and adults ranging in age from 10 to 40 with genetically confirmed FRDA. For 12 weeks, participants were assigned to one of four groups: a control group, NR alone, exercise alone, or exercise plus NR. The exercise program was personalized and done at home and included aerobic workouts on a recumbent trike and resistance training with bands, with remote support from the study team.
The study’s main measurement was peak VO₂, a common test of how well the body uses oxygen during hard exercise – often described as a gauge of heart-and-lung fitness. After 12 weeks, the group that completed both the exercise program and took NR showed a clear, statistically meaningful improvement compared with the control group. The other groups improved a little on average, but not enough to meet the study’s cutoff for a definite difference.
“Overall, our findings support supervised, home-based exercise as a practical and safe way to address a major challenge in Friedreich’s ataxia – low cardiorespiratory fitness,” said Shana McCormack, MD, MTR, the study’s senior author and Scientific Director of the Neuroendocrine Center in the Division of Endocrinology and Diabetes at CHOP. “We’re also encouraged by the idea that pairing a structured behavior program like training with therapies aimed at cellular energy pathways could be a promising direction. That said, we need larger, longer studies to learn whether the benefits last and to clarify whether NR adds meaningful improvement beyond exercise alone.”
No serious adverse events were reported, and side effects were generally mild to moderate, including gastrointestinal symptoms and occasional falls – an important concern in FRDA. Notably, the study did not find that participating in the exercise program increased falls overall compared with groups that did not exercise.
This project was supported by the National Institutes of Health (R01HL149722) and the Friedreich Ataxia Research Alliance support for the CHOP and Penn’s Friedreich's Ataxia Center of Excellence.
Lin 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. Online May 2026. DOI: 10.1016/S1474-4422(26)00082-7.
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Researchers at Children’s Hospital of Philadelphia (CHOP) reported encouraging results of the benefits of exercise for people with Friedreich’s ataxia (FRDA), a rare, inherited condition that gradually affects coordination, strength, and endurance, and can also involve the heart. The findings, reported recently in The Lancet Neurology, showed that a structured, home-based exercise program improved cardiorespiratory fitness over 12 weeks, with the largest gains in participants who paired training with a supplement aimed at supporting cellular energy.
FRDA disrupts the function of mitochondria – the cell’s energy “powerhouses”– and many affected individuals experience significant fatigue and poor exercise tolerance that can impact daily life. Researchers launched this study to address a long-standing gap: although individuals with FRDA are often advised to “stay active,” there has been limited data on what home-based exercise programs are feasible and safe in FRDA, especially given concerns about falls and heart strain.
Additionally, treatment options for these patients are limited. Some individuals with FRDA have experienced benefits from a supplement called nicotinamide riboside (NR), a molecule the body can convert into NAD+, a coenzyme that supports metabolism. However, little was known about its benefits when used alongside home-based exercise programs.
In the study, led by Kimberly Lin, MD, Medical Director of the Cardiomyopathy Program in CHOP’s Cardiac Center, researchers assessed 66 children and adults ranging in age from 10 to 40 with genetically confirmed FRDA. For 12 weeks, participants were assigned to one of four groups: a control group, NR alone, exercise alone, or exercise plus NR. The exercise program was personalized and done at home and included aerobic workouts on a recumbent trike and resistance training with bands, with remote support from the study team.
The study’s main measurement was peak VO₂, a common test of how well the body uses oxygen during hard exercise – often described as a gauge of heart-and-lung fitness. After 12 weeks, the group that completed both the exercise program and took NR showed a clear, statistically meaningful improvement compared with the control group. The other groups improved a little on average, but not enough to meet the study’s cutoff for a definite difference.
“Overall, our findings support supervised, home-based exercise as a practical and safe way to address a major challenge in Friedreich’s ataxia – low cardiorespiratory fitness,” said Shana McCormack, MD, MTR, the study’s senior author and Scientific Director of the Neuroendocrine Center in the Division of Endocrinology and Diabetes at CHOP. “We’re also encouraged by the idea that pairing a structured behavior program like training with therapies aimed at cellular energy pathways could be a promising direction. That said, we need larger, longer studies to learn whether the benefits last and to clarify whether NR adds meaningful improvement beyond exercise alone.”
No serious adverse events were reported, and side effects were generally mild to moderate, including gastrointestinal symptoms and occasional falls – an important concern in FRDA. Notably, the study did not find that participating in the exercise program increased falls overall compared with groups that did not exercise.
This project was supported by the National Institutes of Health (R01HL149722) and the Friedreich Ataxia Research Alliance support for the CHOP and Penn’s Friedreich's Ataxia Center of Excellence.
Lin 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. Online May 2026. DOI: 10.1016/S1474-4422(26)00082-7.
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