Experimental Duchenne Therapy Remains Out of Reach for Most Patients
🤖 AI-generated content — The title and summary were produced automatically by artificial intelligence, without human editorial review.
Brecken, 12, began receiving Avidity Biosciences’ experimental exon-skipping drug last winter and has steadily improved. The treatment enables production of a shortened but functional protein essential to muscle survival and appeared to nearly halt Duchenne muscular dystrophy in some trial patients, but remains inaccessible to most.
Continue in the app — vote & join in ➔Source: STAT News · via ahirlevel.hu