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Wednesday 26th June 2019

Hope for Duchenne muscular dystrophy

25th July 2011

A drug trial has, according to researchers, shown promising results in treating children with Duchenne muscular dystrophy. 


People with the condition have a damaged gene for the protein dystrophin. The trial used a drug as a "molecular scalpel" in order to remove damage and restart dystrophin function.

The condition affects one in 3,500 male babies and causes muscle wastage. This means a child may have to use a wheelchair by the time they reach the age of 10.

It can cause death before the age of 30, when it starts to impact muscles required to breathe and pump blood.

Protein production in the body is directed by genetic code, which in the case of muscular dystrophy is mutated or deleted.

The study, carried out by researchers at the Institute of Childe Health at University College London, attempted to work with the faulty genetic code.

The researchers injected children with antisense RNA, which acted as the "scalpel" and took out part of the genetic code, which was then matched on each end of the mutation.

This formed dystrophin which was not as long but still functioned. Seven of the 19 children in the study showed some dystrophin function after the procedure.

Professor Francesco Muntoni, lead researcher, told the BBC: "The best result was 20% of normal dystrophin levels. That is quite remarkable considering the study was for 12 weeks.

"I've worked with patients with Duchenne muscular dystrophy for many years and this is the first time we can say with confidence that we've made a significant breakthrough towards finding a targeted treatment."


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