Duchenne muscular dystrophy drug shows promising results in GOSH trial

The muscle-wasting condition of Duchenne muscular dystrophy (DMD) has seen a drug trialled which ‘skips over’ a genetic fault for its cause.

The trial of the drug has been led by Great Ormond Street Hospital and has shown ‘promising results.’

Out of 25 children treated with the drug ‘golodirsen’, all showed a significant increase in levels of dystrophin.

Dystrophin is a ‘vital muscle-producing protein’ that DMD patients cannot produce normally.

Due to the lack of the protein, DMD patients end up wheelchair bound by their teens with death occurring in their early 30s.

The medical journal ‘Neurology’ suggests ‘a DNA skipping approach’ can begin to produce dystrophin.

This should slow the progression of the disease.

In golodirsen trials, the drug has proved safe to use on young patients which is suitable for around 8% of them.

Lead researcher and GOSH Paediatric Neurologist Professor Francesco Muntoni said “The success of this study is a significant step towards an effective treatment for children with DMD.”  

“Golodirsen appears to restore dystrophin production in patients with a particular type of genetic mistake, to levels that we think could noticeably slow muscle damage.”

“We must now gather more evidence, but these findings will bolster the application for the drug’s approval in Europe when the time comes.”

Around 100 boys are born with DMD each year.

“The ‘plank’ – known as an antisense oligonucleotide – allows the body to read past the error and produce a shortened version of the protein” according to GOSH.

Professor Francesco went on to say “I hope within a few years that there will be the evidence needed to apply for EMA approval.”

“There are other hurdles after that to expand access for UK patients, but we’re in a very good position to move forward with such positive results from this trial.”