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ENCell’s EN001 Receives Orphan Drug Designation from the U.S. FDA for Charcot-Marie-Tooth Disease
EN001, a novel therapy under development by ENCell, has been granted Orphan Drug Designation by the U.S. FDA for the treatment of Charcot-Marie-Tooth (CMT) disease. Phase 1 clinical trial ...
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FDA Grants Orphan Drug Designation to Revolutionary Cystic Fibrosis Therapy
BOSTON--(BUSINESS WIRE)--Porosome Therapeutics, Inc. (Porosome Therapeutics) today announced the Orphan Drug Designation of its cystic fibrosis therapy by the Food and Drug Administration (FDA).
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NeuroSense’s ALS drug PrimeC shows success in phase 2b, eyes final trial
PrimeC has been given an ‘Orphan Drug’ status by the FDA (Food and Drug Administration) and EMA (European Medicines Agency). PrimeC is a novel therapy with a multi-targeted approach to treat ALS.