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- PMLiVE

DMD

- 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

- PMLiVE

Pfizer buys gene therapy specialist Bamboo

Expands pipeline with Duchenne muscular dystrophy candidate in deal worth $700m

- PMLiVE

Santhera facing long delay for muscular dystrophy drug in US

FDA rejects accelerated marketing application for Raxone to await phase II trial results

Biomarin

BioMarin gives up on muscular dystrophy drug

Makes ‘difficult but necessary’ decision to abandon exon-skipping therapy Kyndrisa

National Institute for Health and Care Excellence NICE logo

NICE backs first Duchenne muscular dystrophy drug

Reverses original decision to reject the therapy in favour of more data

National Institute for Health and Care Excellence NICE logo

NICE backs three hepatitis C meds, turns down Duchenne drug

Benefits of short treatments with high cure rates reinforces drugs value

Biomarin

BioMarin files muscular dystrophy drug in EU

Drisapersen could be the first ‘exon-skipping’ therapy

- PMLiVE

Sarepta seeks new CEO but says eteplirsen is on track

Garabedian will be temporarily replaced by CMO Ed Kaye

- PMLiVE

Sarepta slumps as FDA questions muscular dystrophy drug

US regulator casts doubt on biomarker for eteplirsen

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