Asthma drug improves motor function and strength in children with rare muscle diseases in small trial
· Medical Xpressby Margareta G. Kubista, University of Gothenburg
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Congenital myopathies are a group of rare, inherited muscle diseases that often cause muscle weakness in childhood. This weakness can affect, among other things, the ability to walk, stand up and use one's arms and hands, and in some cases, it can also affect the respiratory muscles.
Currently, there is no cure or pharmacological treatment for these diseases. Care therefore focuses primarily on alleviating symptoms and preventing complications.
Testing an asthma drug for muscle weakness
A study published in eClinicalMedicine investigated whether the substance salbutamol could improve muscle function in children and young people with congenital myopathy. Salbutamol has long been used for asthma, but when administered orally, it also affects skeletal muscle and can, for instance, stimulate the synthesis of muscle protein.
The study included 18 individuals, ages 6–16, with genetically confirmed congenital myopathy. They were randomized to receive either salbutamol in addition to their usual treatment or their usual treatment alone for 24 weeks. They then switched treatments, allowing each participant's results under the two treatments to be compared.
Gains in motor function and breathing
Following the period of salbutamol treatment, the participants' motor function was better than that observed with standard treatment alone. The difference averaged 3.61 points on a standardized motor function rating scale (MFM-32), a change generally regarded as a clear improvement in the context of neuromuscular diseases.
Muscle strength also improved. Grip strength in the dominant hand increased by the equivalent of approximately 1.5 kilograms (3.3 pounds) during salbutamol treatment compared to standard treatment alone. Increased strength was also observed in other muscle groups examined, though these results were not statistically significant.
In addition, lung function improved. On average, forced vital capacity—a measure of the volume of air exhaled after a maximal inhalation—was approximately 0.2 liters higher during salbutamol treatment than during standard treatment.
Improvements across several functional tests were observed in the 11 participants who were able to walk. In a six-minute walk test, the children covered an average distance of 435 meters (1,430 feet) before treatment, whereas with salbutamol, the distance increased to 473 meters (1,550 feet). Children of a similar age typically walk about 600 meters (1,970 feet) in six minutes.
The researchers also assessed how quickly the ambulatory children in the study could rise from the floor to a standing position. On average, it took them 8.29 seconds to stand up, but with salbutamol, that time was reduced to 6.39 seconds. A child with normal strength rises from the floor to a standing position in 1–2 seconds.
Larger studies needed to confirm benefits
The treatment was well tolerated, and no serious side effects were reported.
"The results suggest that oral salbutamol could potentially become a symptom-relieving treatment option for children and adolescents with congenital myopathies in the future, pending the development of other drugs. However, these findings need to be confirmed in larger international studies involving longer treatment periods and follow-up," says Eva Michael, a doctoral student in pediatric neurology at the University of Gothenburg and a specialist physician at the Queen Silvia Children's Hospital.
"Simply the fact that they had more energy, that they could manage a full day at school followed by activities and homework without becoming completely exhausted and bursting into tears, is very important for these children," says Michael.
Publication details
Eva Michael et al., Effects of oral salbutamol treatment on motor function and muscle strength in congenital myopathies (COMPIS): a single-centre, randomised, open-label, blinded-endpoint, crossover trial in Sweden, eClinicalMedicine (2026). DOI: 10.1016/j.eclinm.2026.104186
Journal information: EClinicalMedicine
Key medical concepts
AlbuterolMotor functionMuscle Strength
Clinical categories
PediatricsNeurology Provided by University of Gothenburg Who's behind this story?
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