Asthma Medication Improves Motor Skills and Strength in Children With Congenital Myopathies

A common asthma medication improves motor skills and muscle strength in children with congenital myopathies, a group of rare, inherited muscle diseases that currently have no pharmacological cure, News-Medical reported. In a randomized crossover trial, patients receiving oral salbutamol were able to walk further, grip tighter, and stand up from the floor more quickly.

Trial Design and Patient Outcomes in Sweden

The study, known as the COMPIS trial and published in the Lancet journal eClinicalMedicine, investigated whether salbutamol could improve muscle function in children and young people. Researchers at Queen Silvia’s Children’s Hospital in Gothenburg and nationwide neuromuscular centers enrolled 18 participants aged 6 to 16 with genetically confirmed congenital myopathy, according to reporting from Mirage News and ScienMag. The cohort included individuals with variants in genes such as RYR1, ACTA1, NEB, SELENON, TPM2, TPM3, and MYBPC1.

Participants were randomized to receive either 24 weeks of daily oral salbutamol alongside their usual care or standard care alone before crossing over to the alternative treatment. ScienMag noted that physiotherapists and occupational therapists performing assessments were masked to treatment assignment. Following the treatment period, participants’ motor function scored an average of 3.61 points higher on the standardized MFM-32 rating scale compared to standard treatment alone, while ScienMag reported a mean rise of 3.72 points.

Grip Strength and Respiratory Capacity Changes

Muscle strength metrics also shifted during the trial. Dominant hand grip strength increased by the equivalent of approximately 1.5 kilograms during salbutamol treatment. Researchers observed increased strength in other muscle groups, though those specific results lacked statistical significance. Lung function similarly registered gains, with forced vital capacity averaging approximately 0.2 liters higher during salbutamol treatment than during standard care.

Among the eleven participants able to walk, performance in a six-minute walk test increased from an average baseline distance of 435 meters to 473 meters. By comparison, children of a similar age typically walk about 600 meters in six minutes. Ambulatory participants also reduced the time required to rise from the floor to a standing position from an average of 8.29 seconds down to 6.39 seconds, while children with normal strength typically complete the movement in one to two seconds.

Daily Energy Gains and Medical Context

Beyond clinical metrics, investigators highlighted practical improvements in daily endurance. 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, said Eva Michael, a doctoral student in pediatric neurology at the University of Gothenburg and a specialist physician at Queen Silvia Children’s Hospital.

Because no cure exists, current care focuses on managing symptoms through physiotherapy.

Future International Studies and Safety Monitoring

The treatment was well tolerated, and no serious side effects were reported during the 24-week intervention period. 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, Michael stated.

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