FDA grants rare pediatric disease designation to alpha kinase inhibitor for ROSAH syndrome

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.
The rare pediatric disease designation is granted by the FDA for a serious or life-threatening condition that affects fewer than 200,000 people in the United States and primarily affects those aged younger than 18

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