Experimental drug helps some Duchenne patients while others remain untreated
Two boys in Minnesota and San Diego show different outcomes with an experimental treatment for Duchenne muscular dystrophy.
Different results for similar patients
One boy named Brecken started receiving the medicine last winter. His condition has improved steadily while another boy named Yannick is declining quickly. The two boys play video games together but rarely discuss their specific medical situations.
Treatment targets specific gene mutations
The drug developed by Avidity Biosciences allows cells to skip part of the faulty gene. This process creates a shortened version of a protein needed for muscle survival. Trials suggest this approach could help about 70 percent of patients in the United States.
Access remains limited for most
The current medicines must be tailored to each patient specific mutation. This requirement keeps the therapy out of reach for many families. Only a fortunate few can access this promising treatment right now.
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