Boys with Duchenne muscular dystrophy are keeping their arm strength longer on a new therapy

For boys with Duchenne muscular dystrophy — a rare disease that weakens every muscle and for which heart failure is the leading cause of death — small victories are everything. New data presented October 5 at the World Muscle Society Congress in Hiroshima show that patients on the cell therapy Deramiocel kept their upper-limb function improving well beyond the first year, in the open-label extension of the HOPE-3 Phase 3 trial of 106 patients (whose one-year results were published in The Lancet in July). With no cure for the disease, which affects about 15,000 people in the US, mostly boys, a therapy that preserves the strength to lift an arm is hope measured in daily life.