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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.

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.

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. 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.

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