“My friend Yannick is waiting to get the next Avidity therapy,” 12-year-old Brecken wrote in a letter to Novartis. “He was so happy for me when I got it. It is getting hard for him to throw the ball for his dog Rosie and drink a glass of water. This makes me sad.” www.statnews.com/2026/10/07/m...
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In Duchenne muscular dystrophy, a promising therapy is available to a fortunate few
Exon-skipping drugs present a critical test for the FDA’s goal of encouraging treatments for rare disease.