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- PMLiVE

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- PMLiVE

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

- PMLiVE

FDA approves Sarepta rival NS Pharma’s Duchenne drug

Drug approved for patients with exon 53 mutation

Roche Basel Switzerland

Roche pays $1.15bn for Sarepta’s Duchenne gene therapy

Pens another gene therapy deal following takeover of Spark Therapeutics

- PMLiVE

Sarepta rival NS Pharma files Duchenne drug with FDA

Another blow for Sarepta following FDA rejection earlier this year

- PMLiVE

FDA rejects Sarepta’s new Duchenne drug over safety fears

FDA gave no warnings of concerns, says chief exec

- PMLiVE

Sarepta investigating mistakenly submitted Duchenne therapy report

Study participant fell seriously ill

- PMLiVE

Duchenne UK backs Evox dystrophin delivery project

Funding will drive preclinical studies on dystrophin-bearing exosomes

- PMLiVE

Daily Brief: Sarepta deepens gene therapy investment, NICE changes mind on Besponsa, Digital Medicine company Akili raises $13m

News from pharma, biotech and healthcare

- PMLiVE

Sarepta soars on positive Duchenne data

Trial results position Sarepta as a “leader in the field” according to analysts

- PMLiVE

FDA reviewer unimpressed with PTC’s Translarna

Finds the muscular dystrophy treatment's evidence to be 'not persuasive'

- PMLiVE

Sarepta eyes FDA filing for DMD drug golodirsen after positive trial

The exon-skipping drug raised functional dystrophin levels

- PMLiVE

Sarepta soars as FDA clears its muscular dystrophy drug

Analysts anticipate blockbuster status for DMD therapy despite efficacy concerns

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