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Pros and Cons, Orphan Drug Act – Flash Briefing
The way I understand it, the Orphan Drug Act has benefited rare disease patients like us, by making it more enticing for pharmaceutical companies to develop drugs that otherwise wouldn’t be done. The only FDA medications specifically approved for LEMS, Ruzurgi and Firdapse, may not have become generally available to our population without the ODA.
Here’s a Flash Briefing about the Orphan Drug Act.
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