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Drug Farm Announces U.S. FDA Orphan Drug Designation for DF-003 for the Treatment of ROSAH Syndrome
ALBANY, N.Y. & SHANGHAI--(BUSINESS WIRE)--Drug Farm today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to DF-003, the company’s investigational small molecule inhibitor targeting ALPK1, for the treatment of patients with ROSAH syndrome, a rare, genetically driven systemic autoinflammatory disease with significant unmet medical need. ROSAH syndrome (Retinal dystrophy, Optic nerve edema, Splenomegaly, Anhidrosis, and Headache) is a progressi