Duchenne Muscular Dystrophy (DMD)

DMD Research: Another Dystrophin Stand-In?

Delivering the gene for the missing dystrophin protein to the muscles of children and young men with Duchenne muscular dystrophy (DMD) is one of several therapeutic avenues under intense investigation in this disease. However, the strategy has technical drawbacks, and some experts worry that introduction of a previously absent protein could provoke a dangerous immune response.

CMD Research: Promising Leads

Recently published findings from two independent groups have suggested possible treatment pathways for the merosin-deficient and integrin-deficient forms of congenital muscular dystrophy (CMD).

Doxycycline fights cell death and lessens disease severity in merosin-deficient mice

Speeding the Course of Clinical Trials

Trial Results in DMD Heralded as 'Proof of Concept'

The Dec. 27 issue of the prestigious New England Journal of Medicine featured encouraging results of a phase 1 clinical trial of an exon skipping compound in four boys with Duchenne muscular dystrophy (DMD). Preliminary findings from this trial, conducted in the Netherlands, were announced in May (see Progress Reports).

First US Trial of DMD Gene Therapy Under Way

The first U.S. human gene therapy trial directed at Duchenne muscular dystrophy (DMD) was launched yesterday at Columbus (Ohio) Children's Hospital, the Muscular Dystrophy Association (MDA), Children’s Hospital, and Asklepios Biopharmaceutical Inc. (AskBio) announced today. 

Neurologist Jerry Mendell administered an injection of AskBio’s Biostrophin, which contains a functional gene for the muscle protein dystrophin, into the biceps of Andrew Kilbarger, 8, of Lancaster, Ohio.

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