NS Pharma
Sarepta Therapeutics’ DMD gene therapy shows early success
Most patients with Duchenne muscular dystrophy require full-time use of a wheelchair by their early teens
FDA approves Sarepta rival NS Pharma’s Duchenne drug
Drug approved for patients with exon 53 mutation
FDA sets Q3 decision date for NS Pharma’s DMD drug
If approved it will become second-to-market after Sarepta
Roche pays $1.15bn for Sarepta’s Duchenne gene therapy
Pens another gene therapy deal following takeover of Spark Therapeutics
Sarepta rival NS Pharma files Duchenne drug with FDA
Another blow for Sarepta following FDA rejection earlier this year
FDA rejects Sarepta’s new Duchenne drug over safety fears
FDA gave no warnings of concerns, says chief exec
Sarepta investigating mistakenly submitted Duchenne therapy report
Study participant fell seriously ill
Daily Brief: Sarepta deepens gene therapy investment, NICE changes mind on Besponsa, Digital Medicine company Akili raises $13m
News from pharma, biotech and healthcare
Sarepta soars on positive Duchenne data
Trial results position Sarepta as a “leader in the field” according to analysts
Sarepta eyes FDA filing for DMD drug golodirsen after positive trial
The exon-skipping drug raised functional dystrophin levels
Sarepta soars as FDA clears its muscular dystrophy drug
Analysts anticipate blockbuster status for DMD therapy despite efficacy concerns
Sarepta seeks new CEO but says eteplirsen is on track
Garabedian will be temporarily replaced by CMO Ed Kaye