23 April 2007

The science journal 'Nature' today published research which has promising results for some forms of Duchenne muscular dystrophy. The drug, PTC124, is in trial stage at the moment and has been developed in the form of a daily pill.

The Muscular Dystrophy Campaign's Director of Research, Dr. Marita Pohlschmidt, said “The findings described in the Nature article by Professor H. Lee Sweeney and colleagues are encouraging, particularly in the light of the clinical studies which are being carried out in the United States."

"This drug appears to have the potential to become a treatment in the longer term for some people with Duchenne muscular dystrophy. The Muscular Dystrophy Campaign is looking forward to the publication of the full results of these clinical trials so that we can see more clearly what the prospects and possible timescales are for treatment.”

The Muscular Dystrophy Campaign's Research Department has produced a summary of this research and answers some key questions. Click here to read more.

4 May 2007 – Update
At the annual meeting of the American Academy of Neurology on 4 May 2007 PCT Therapeutics presented interim Phase 2 results of PCT124 in boys with Duchenne Muscular Dystrophy due to a nonsense mutation. The trial has been conducted at three different centres in the US and the company claims that overall the results are positive.

In this Phase 2 study three cohorts of boys have received three different dose levels of PCT124 over a period of 28 days. The two lower dosage groups (26 patients) have been analysed and the results show that treatment with PCT124 is associated with increased production of dystrophin in the muscle and a reduction of the creatinine kinase levels in the blood in at least 50% of evaluated boys. The analysis of the higher dose level is still ongoing.

Several parents and teachers also reported that boys treated with PCT124 showed improvements in their activity level and increased endurance.
The MDC Research Team will continue to monitor developments and will provide updates when new information becomes available.