Ahead of FDA, EMA rec­om­mends au­tho­riz­ing new gene ther­a­py treat­ment for ul­tra-rare dis­ease

Aro­mat­ic amino acid de­car­boxy­lase (AADC) de­fi­cien­cy is an ul­tra-rare ge­net­ic dis­ease that leaves pa­tients un­able to pro­duce cer­tain hor­mones in the brain, such as dopamine …

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