ENDPOINTS NEWS54m
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 ...