Pharmafile Logo
T40 2022 Cover
Diversity, Equity and Inclusion
Communiqué 53 Cover Image
- PMLiVE

AAVrh74.MHCK7

- 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

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

Subscribe to our email news alerts

Latest jobs from #PharmaRole

Latest content

Latest intelligence

Quick links