Re­gen­eron’s piv­otal tri­al for an ul­tra-rare dis­ease fails the pri­ma­ry — but don't count them out at the FDA

Re­gen­eron’s po­ten­tial­ly piv­otal study for a new drug to treat an ul­tra-rare and lethal bone dis­or­der failed the pri­ma­ry end­point — but the R&D team thinks they can still get the ball in­to the reg­u­la­to­ry end zone with the pos­i­tive da­ta they have on hand.

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