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MDA giving $2.5M for gene therapy trial

The Muscular Dystrophy Association is donating $2.5 million to help fund the second stage of a gene therapy trial aimed at finding a cure for Duchenne Muscular Dystrophy, the association said Tuesday.

A matching grant by Asklepios BioPharmaceutical Inc. in Chapel Hill, N.C., will enable researchers to test the safety and effectiveness of Biostrophin, the company’s key gene therapy candidate to treat DMD.

Patients with DMD are primarily males who are afflicted in childhood. The disease causes a loss of muscle strength and leads to death from cardiac or respiratory lung failure, said MDA spokesman Bob Mackle.

The gene therapy, using nanotechnology, would replace a muscle protein, dystrophin, that does not exist in patients with DMD.

Mackle said the grant to the Chapel Hill pharmaceutical company is the largest awarded by the nonprofit MDA to a for-profit company.

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