Pioneering Duchenne drug approved for use on NHS Scotland

DMD is a rare genetic condition which causes progressive muscle weakness, eventually impacting the heart and lungs.

It is typically diagnosed in boys between the ages of 3 and 6.

While there is no cure, advocates for people living with the condition believe that givinostat can slow the progression of the disease, offering families precious time with their children.

The medicine, which was approved by the Medicines and Healthcare products Regulatory Agency (MHRA), blocks histone deacetylase - which is an enzyme that is overactive in DMD and inhibits muscle repair.

Emily Reuben OBE, the co-founder and chief executive of Duchenne UK, said that the approval was a 'significant breakthrough' for people living in Scotland.

She remarked: "In the last year, we've gone from having extremely limited treatment options available to now having two approved medicines, offering hope to hundreds of families, and in the case of givinostat, the promise of slowing down the progression of this devastating disease."

Reuben added that the drug must now be approved by NHS England and made available across the UK.

She added: "We also continue to explore how patients who've already lost their ability to walk can be given the opportunity to potentially benefit from this new treatment."

Families in Scotland formed a united front to demand action in early 2025. They met Health Secretary Neil Gray, who pledged to speak with NHS officials to ensure the rollout of the drug.

While health boards cited logistical hurdles, they eventually committed to early access programmes and began contacting DMD families to arrange treatment.

Reflecting on the drug's approval, Andy Fletcher, Chief Executive of Muscular Dystrophy UK noted: "This is a landmark moment for families in Scotland. People with muscle wasting conditions, like Duchenne muscular dystrophy, don't have time to waste.

"Accessing treatments as early as possible can make a significant difference and with givinostat now accepted for NHS