The FDA changes the rules — rare disease patients pay with their lives

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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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