NHS Choices has conducted an assessment of a report by the BBC that a new study has provided “hope for Duchenne muscular dystrophy.”

The condition results from genetic mutations that prevent the body from producing the regular form of a protein called dystrophin, which is essential for forming vital connections in the muscle tissue. The UK study, funded by the UK Medical Research Council and AVI BioPharma, the manufacturer of the drug being tested, was published in The Lancet. It tested a new drug known as AVI-4658 designed to make the body bypass genetic mutations when producing dystrophin. The drug was given to 19 children and the higher doses led to an increase in dystrophin. Researchers believe that drugs which are designed to make the body “skip over” mutations in this way could be used to treat approximately 83% of Duchenne muscular dystrophy cases. However, the drug used in this trial only targeted mutations in a region implicated in 13% of cases.

The assessment notes that the BBC covered the story well and that the study was well conducted and demonstrated the potential of this approach for increasing the levels of dystrophin in the short term. However the principal aim of the study was to establish appropriate dosages of the drug, therefore its safety profile and efficacy will need to be confirmed in larger, longer-term studies.