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Opinion: Capricor’s Duchenne muscular dystrophy drug and the countless tiny decisions that can make or break a study
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关键摘要
Five years ago, we moved my son into a college dorm.I know that makes us sound like thousands of other families with 18-year-olds, but my son has Duchenne muscular dystrophy.…
- He couldn’t walk, and because of his waning arm strength, we all thoug…
- Instead, we are now getting ready to move him into that same dorm agai…
- He has spent those five years on Capricor’s deramiocel, a drug that re…
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正文提要
Five years ago, we moved my son into a college dorm. I know that makes us sound like thousands of other families with 18-year-olds, but my son has Duchenne muscular dystrophy. He couldn’t walk, and because of his waning arm strength, we all thought his college independence would be short-lived.
Instead, we are now getting ready to move him into that same dorm again, where he still lives independently, so he can begin the second year of his master’s degree study. He has spent those five years on Capricor’s deramiocel, a drug that received a negative FDA advisory committee vote last week on its secondary outcome: the definitiveness of its ability to stabilize heart function in a population of boys that included ones whose hearts were still stable. It was much noisier data than the significance seen in the subpopulation that already had signs of heart dysfunction.