Scalpel treatment for Duchenne muscular dystrophy

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A drug trial involving 19 children has been carried out to investigate the use of a molecular scalpel in treating Duchenne muscular dystrophy. The cond...

A drug trial involving 19 children has been carried out to investigate the use of a molecular scalpel in treating Duchenne muscular dystrophy.

The condition, which affects 1 in 3,500 newborn boys, is caused by a damaged gene for the protein dystrophin. Sufferers’ muscles waste away with the result that patients may be in a wheelchair by the age of 10. By the age of 30 the condition can become life-threatening, affecting the muscles required to breathe and pump blood around the body.

The study, which was published in the Lancet, involved the use of a molecular scalpel to remove the damage in the gene and to restore dystrophin production.

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Seven of the 19 children experienced some degree of restored dystrophin production. The most promising result was 20% of normal dystrophin levels.

Researchers noted that this technique would only be appropriate for this specific mutation, which affects 13% of sufferers. Alternative treatments would need to be found for patients with other types of mutations.

However, the team were encouraged by the results of the study, which is a major breakthrough in many years of seeking a targeted treatment for the disorder.

The Muscular Dystrophy Campaign also praised the trial.

Dr Marita Pohlschmidt, director of the cause, said: "If we can change severe symptoms in Duchenne into something milder, that would be fantastic.

"We have fought to find a treatment for this devastating condition for the past 50 years. Today we can say with real confidence that we're going to win that battle. Parents of these boys can have real hope for the future."

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