FDA ap­proves PTC’s ul­tra-rare dis­ease gene ther­a­py for broad­er pa­tient pop­u­la­tion than Eu­rope

The FDA has cleared PTC Ther­a­peu­tics’ gene ther­a­py for AADC de­fi­cien­cy, an ul­tra-rare dis­ease that im­pacts the nerve cells and brain.

The gene ther­a­py was …

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