GENE ONLINE|News &
The FDA Granted These Three Companies Orphan Drug Designations in One Day
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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2024 Biomedical Final Pitch Competition
Room DA1620, Dana Building, Dana-Farber Cancer Institute, 99 Jimmy Fund Way, Boston, MA 02115
