BioSpace

FDA's rare disease framework faces its first real test in Capricor’s DMD drug

Last month, the FDA committed to designing a "fit-for-purpose" framework for regulating rare disease therapies based on patient need and realistically attainable evidence. With the upcoming decision for Capricor’s Duchenne muscular dystrophy cell therapy deramiocel, this paradigm faces its first key test.

By BioSpace

1 min read
FDA's rare disease framework faces its first real test in Capricor’s DMD drug
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