Duchenne Muscular Dystrophy: PTC124 Daily Pill Research 'Encouraging'

Main Category: Muscular Dystrophy / ALS
Article Date: 26 Apr 2007 - 12:00 PDT

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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."

Our research department will publish a summary of the research here shortly.

The magazine, 'Nature', (website is http://www.nature.com) is subscription only so the original article is not freely available. The article itself is written in medically technical language with no summary in layman's terms. If you'd like to know more, please return to our website later to find the summary drawn up by our research department.

Muscular Dystrophy Campaign

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