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Deramiocel (CAP-1002)

Duchenne muscular dystrophy

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92 sources

Product information and disease descriptionPlace of product in therapyDisease descriptionBurden of Duchenne muscular dystrophy

Humanistic burden and health-related quality of life

5 evidence topics · 6 sources

Humanistic burden: Chilean cross-sectional study

“Compared with controls, patients reported markedly lower physical functioning (15% vs. 100%) and general health (62% vs. 82%)” (opens the source at this quote in a new tab)

Health-state utilities

“Both patient and caregiver utilities trended lower with higher disease severity. Variability in utilities was observed based on instrument, respondent type, and country.” (opens the source at this quote in a new tab)

Health-state utilities: upper-limb function in non-ambulatory DMD

“Utility values for less severe non-ambulatory health states described by level of upper limb function are novel.” (opens the source at this quote in a new tab)

DMD-QoL and HUI-2 utility estimates

“DMD-QoL utilities did not vary meaningfully across health states compared with HUI-2 scores, which tended to decline with deteriorating mobility.” (opens the source at this quote in a new tab)

Caregiver anxiety and depression

“Half of all caregivers (383 of 770) reported being moderately or extremely anxious or depressed” (opens the source at this quote in a new tab)