Expert Interview
Discussing Deramiocel for cardiomyopathy in DMD patients, the HOPE-3 data, and approval/commercial uptake expectations
Ticker(s): CAPRInstitution: Rady Children's Health
- Associate Clinical Professor of Pediatric Cardiology
- Clinical and research interests in infants, children, and adolescents with cardiomyopathy,, heat failure and heart transplantation
- Currently care for over 1000 children with cardiomyopathy and/or heart failure
how many DMD-Cardiomyopathy patients do you manage?
Added By: wilson_adminHow convincing is the HOPE-3 data to you?
Added By: wilson_adminAssuming approval, how many of your DMD patients would you attempt to get on this therapy?
Added By: wilson_adminIs 1.2 points on the PUL-2 clinically meaningful to patients given it is a 42 point scale?
Added By: clairecipolla1The trial enrolled n=106 yet the LVEF data only reported on n=83 patients. Some patients were apparently not included due to scan quality, missing scans or inability to undergo cMRI. There was an imbalance between the number of patients excluded from the treatment arm vs placebo. How might this impact the statistical analysis and would this data be MNAR which could impact the p value. Does it seem logical that so many patients would have missing scans? Also, note, the endpoint reported on was not absolute mean change but rather mean ranked change. Is this an appropriate way to report LVEF?
Added By: clairecipolla1Are You Interested In These Questions?
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