healio.com
The FDA has granted rare pediatric disease designation to an alpha-kinase 1 inhibitor to treat patients with retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis and headache, or ROSAH, syndrome.According to a press release from developer Drug Farm, DF-003 is currently being evaluated in a phase 1 clinical trial assessing safety and pharmacokinetics in healthy volunteers.
11 months ago