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• Sarepta eyes FDA filing for DMD drug golodirsen after positive trial

  normal from an average baseline level of 0.095% of normal, which is a 10-fold increase and superior to that seen with exon 51-targeting Exondys 51 (eteplirsen).

• FDA approves first steroid for Duchenne muscular dystrophy

  eteplirsen). "For the first time, patients in the US with Duchenne will have widespread access to an FDA approved medicine that is indicated for all genetic forms of the condition," said

• Sarepta soars as FDA clears its muscular dystrophy drug

  Sarepta shares closed up 74% yesterday after news of the conditional approval for Exondys 51 (eteplirsen) broke - taking investors by surprise. ... in the EU for another exon 51-skipping therapy - BioMarin's now-abandoned drisapersen - could block

• Santhera facing long delay for muscular dystrophy drug in US

  The US agency has also asked for new data on Sarepta's exon-skipping drug eteplirsen that has extended the already-delayed review beyond the new deadline of 26 May, although

• The death of deference

  The recent news that the FDA has delayed its decision about Sarepta Therapeutic's eteplirsen did just that. ... But the eteplirsen situation is a particularly strong illustration of one of the less discussed forces shaping our industry, one whose