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Duchenne Muscular Dystrophy: New Analysis Shows Drug Slows Down Respiratory Decline


Duchenne muscular dystrophy occurs in boys and is characterized by progressive muscle degeneration and weakness leading to a decline in respiratory function. Strategies to arrest this severe progressive deterioration are needed to extend lives and improve quality of life. Results of three clinical trials using eteplirsen, an exon-skipping antisense oligonucleotide, show promising results, according to a study published in the Journal of Neuromuscular Diseases.

Muscular dystrophy is a group of genetic disorders that results in increasing weakening and breakdown of skeletal muscles. Near absence of dystrophin, a critical protein, results in inflammation, necrosis, and eventual replacement of functional muscle tissue with fibrosis and fat.

Duchenne Muscular Dystrophy: New Analysis Shows Drug Slows Down Respiratory Decline