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2022-05-13| Trials & Approvals

The FDA Granted These Three Companies Orphan Drug Designations in One Day

by Reed Slater
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May 12 was a big day for advancing rare disease treatment as the FDA granted Orphan Drug Designation to Stealth BioTherapeutics, Neurocrine Biosciences, and Editas Medicine. Stealth BioTherapeutics aims to treat Duchenne muscular dystrophy, Neurocrine Biosciences is focused on chorea associated with Huntington’s disease, and Editas Medicine on beta thalassemia, a rare blood disorder.

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