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Orphan Diseases
News
May 22, 2024
1 min read
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FDA grants orphan designation to Fragile X syndrome therapeutic
The FDA has granted orphan drug designation to a small molecule therapeutic for individuals with Fragile X syndrome.
News
January 31, 2024
2 min watch
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VIDEO: Drug-eluting contact lenses could provide alternative to eye drops
WAILEA, Hawaii — In this Healio Video Perspective from Hawaiian Eye 2024, Cynthia Matossian, MD, FACS, discusses how a soft contact lens may deliver medication to the front of the eye as an alternative to eye drops.
News
August 20, 2021
1 min read
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Paratek receives orphan drug status for Nuzyra to treat NTM lung disease
The FDA granted Paratek Pharmaceuticals orphan drug designation for Nuzyra to treat infections caused by nontuberculous mycobacteria, the company announced this week.