Year in Review 2025 (2)-combined - Flipbook - Page 11
muscular dystrophy
Duchenne Muscular Dystrophy (DMD) is a severe,
X-linked genetic disorder characterized by progressive
muscle degeneration affecting the skeletal, respiratory,
and cardiac muscles. It is caused by the absence of
functional dystrophin, a key structural protein in muscle
cells. DMD affects approximately 15,000 individuals in
the United States and primarily impacts boys. Over time,
deterioration of the heart muscle leads to cardiomyopathy and heart failure, which is the leading cause
of death in DMD. There is no cure, and treatment options
remain limited.
HOPE-3 is a Phase 3, multi-center, randomized,
double-blind, placebo-controlled clinical trial consisting
of two cohorts evaluating the safety and efficacy of
Deramiocel in participants with DMD. Non-ambulatory
and ambulatory boys who meet eligibility criteria are
randomly assigned to receive either Deramiocel or
placebo every 3 months for a total of four doses during
the first 12 months of the trial. A total of 106 eligible
subjects were randomized in the dual-cohort trial.
