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This Small Molecule Protects the Muscles of Patients With Duchenne Muscular Dystrophy –

Summit Therapeutics’ treatment for the devastating disease, Duchenne muscular dystrophy, reduces inflammation, which could protect muscle fibers from damage.

Summit Therapeutics focuses on the development of treatments that can help patients suffering from diseases that currently lack options. The biotech has reported Phase II results for its drug for the genetic disease, Duchenne muscular dystrophy (DMD). Summit’s candidate, ezutromid, significantly reduced muscle inflammation, which should, in turn, reduce damage to muscle fibers.

DMD, a progressive muscle wasting disease, affects around 50,000 boys and young men in the developed world. The disease is caused by mutations in the gene that encodes dystrophin, a protein required for keeping muscles healthy and functioning correctly. Muscle weakness begins in the hips, pelvis, thighs, and shoulders, then spreads to the arms and legs, and eventually reaches the heart and muscles around the lungs. With no cure for the disease available, patients with the disease often die before the age of 30.

Summit believes that the modulation of another protein, utrophin, could offer a treatment for DMD. Utrophin is functionally and structurally similar to dystrophin and its presence improved muscle performance in preclinical studies. Summit’s drug, ezutromid, is a utrophin modulator that replaces missing dystrophin to maintain muscle fibers. The drug has received Orphan Drug, Fast Track, and Rare Pediatric Disease designations from the FDA.

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