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FDA panel concludes Translarna data inconclusive in DMD

September 28, 2017 11:42 PM UTC

FDA's Peripheral and Central Nervous System Drugs Advisory Committee voted overwhelmingly that efficacy data for Translarna ataluren to treat nonsense mutations in Duchenne muscular dystrophy (DMD) were inconclusive.

Ten of 11 panel members voted that although it is possible Translarna from PTC Therapeutics Inc. (NASDAQ:PTCT) may be effective, more work is needed to establish whether the small molecule that facilitates complete translation of proteins containing nonsense mutations is effective. One member, patient representative Jeffrey Watkins, voted that the data were sufficient to conclude Translarna is effective...

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