The Food and Drug Administration just dealt a crushing setback to patients living with Duchenne muscular dystrophy.

Last week, the FDA’s advisory committee voted 9–3 that the evidence was insufficient to establish the effectiveness of Deramiocel, Capricor Therapeutics’ treatment for a heart condition associated with DMD. That decision will stand, and patients will suffer and die, unless it is reversed by Aug. 22. 

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I have spent my career working across bioscience, clinical development, and medicine. I have seen therapies once regarded as experimental become standards of care, saving or extending lives. That progress was possible because researchers could test their hypotheses against clear scientific requirements. Innovation depends on predictable standards, and patients lose when developers cannot understand the approval pathway.

For years, opponents of “right to try” warned that loosening the rules for rare-disease therapy approvals would turn drug........

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