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Hope for Duchenne muscular dystrophy patients by new drugs

Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness. It is one of nine types of muscular dystrophy.

DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. Symptom onset is in early childhood, usually between ages 3 and 5. The disease primarily affects boys, but in rare cases it can affect girls.

Muscle weakness can begin as early as age 3, first affecting the muscles of the hips, pelvic area, thighs and shoulders, and later the skeletal (voluntary) muscles in the arms, legs and trunk. The calves often are enlarged. By the early teens, the heart and respiratory muscles also are affected.

The new drug has been approved by the FDA for the treatment of DMD. It is an “exon skipping antisense oligonucleotide” that modulates the genetics of the DMD patients to slow the progression of their disease.

This study was led by Dr. Navid Z. Khan, Senior Director, Global Medical Affairs, Sarepta Therapeutics, Inc., Cambridge, MA, USA. He and his team assessed the effect of a new drug on respiratory functions in DMD patients from three clinical trials and compared it with patients from Cooperative International Neuromuscular Research Group Duchenne Natural History Study (CINRG DNHS) global database.

Data from more than 400 DMD patients were divided into three cohorts, of which 13 percent have typical DMD mutations. These mutations can be treated by the exon 51 skipping therapy, explain the researchers.(source)

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