The FDA has cleared an investigational new drug application for an alpha-kinase 1 inhibitor for clinical evaluation in those with retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis and headache, or ROSAH, syndrome. Drug Farm, manufacturer of DF-003, said in a press release that the first-in-class oral therapeutic will be examined in a pending clinical trial to examine safety, efficacy
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FDA grants rare pediatric disease designation to alpha kinase inhibitor for ROSAH syndrome
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