Deramiocel Significantly Slows Upper Limb Functional Decline in Duchenne Muscular Dystrophy: Skeletal Muscle Outcomes from the Phase 3 HOPE-3 Trial (LS2.003)

Key Points

To confirm the musculoskeletal efficacy of deramiocel in individuals with Duchenne muscular dystrophy (DMD).
Phase 3 trial evaluating deramiocel's effects on upper limb function.
Conducted assessments both in clinic and at home.
Deramiocel significantly slows decline in upper limb function in DMD.
Efficacy measures showed substantial improvement in musculoskeletal outcomes.

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Demonstrates improved musculoskeletal outcomes in Duchenne muscular dystrophy, suggesting potential for functional preservation.

Confirmation of musculoskeletal efficacy of deramiocel in DMD using clinic and home-based assessments

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Iannaccone et al. (2026) studied this question.

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