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Drug Farm Receives Rare Pediatric Disease Designation from the U.S. FDA for DF-003 to Treat ROSAH Syndrome
GUILFORD, Conn.--(BUSINESS WIRE)--Drug Farm, a clinical-stage biopharmaceutical company announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) Designation for the company’s alpha-kinase 1 (ALPK1) inhibitor, DF-003, to treat patients with ROSAH (retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis and headache) Syndrome. DF-003 is currently being evaluated in a Phase 1 trial (NCT05997641) to assess safety and pharmacokinetics in normal healt