No effective treatment for muscular dystrophy

Hyderabad, Nov 27 (UNI) ''Although much is now understood about the pathogenesis of Duchenne Muscular Dystrophy (DMD) there is still no effective treatment'', Kay E Davies, Honorary Director of MRC Functional Genetics Unit, Oxford, UK, said.

Delivering the Foundation day lecture on 'Duchenne Muscular Dystrophy- Past, Present and Future', organised by Centre for Cellular and Molecular Biology (CCMB) here yesterday, Professor Davies said several approaches were being actively pursued on DMD, but the problem is a challenging one, since dystrophin is a large structural protein and there was a need to deliver it to all muscles, including diaphragm and heart muscle.

She said DMD was a devastating X-linked progressive muscle wasting disease, affecting approximately one in 3,500 boys. Patients were usually confined to a wheelchair before the age of twelve and die in their late teens or early twenties, usually of respiratory failure, she added.

Before the advent of DNA recombinant technology, carrier detection for the disease was unreliable and parental diagnosis was not possible, she said.

Prof Davies said recent advances suggests there was cause for optimism, as significant progress was being made.

The substantial progress made in the understanding of DMD and the resulting improvement in patient's lives was a paradigm for the study of other genetic disorders, a release said here.

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