Latest news

• Daily Brief: ASCO latest, Europe cautions on immunotherapy, rejects Duchenne treatment

  Europe still unconvinced by Duchenne treatment Exondys. Finally, the CHMP has confirmed its earlier decision to refuse marketing authorisation for Sarepta’s Exondys(eteplirsen).

• FDA reviewer unimpressed with PTC’s Translarna

  that the firm was emboldened by the FDA’s approval last year of another DMD therapy from Sarepta - Exondys 51 (eteplirsen) - after Janet Woodcock, head of the agency’s Center for

• 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