An experimental gene therapy developed by Lexeo Therapeutics has shown early promise in treating one of the most serious complications of the rare neurodegenerative disorder, Friedreich's a
After getting a green light from the European Commission, Biogen’s Skyclarys is the first approved medicine for the inherited neurological disease Friedreich’s ataxia (FA)
Biogen’s Skyclarys is on course to becoming the first approved medicine for the inherited neurological disease Friedreich’s ataxia (FA) in the EU after it was recommended
Biogen has turned to M&A to offset its recent troubles with pipeline failures and aborted product launches, agreeing to a $7.3 billion deal to acquire Reata Pharma.
PTC Therapeutics has cued up no fewer than three clinical trial readouts this quarter that could lead to new regulatory filings – but the latest for vatiquinone in inherit
Motion capture technology used to bring fantasy characters to life in movies like Avatar and The Lord of the Rings is being used alongside artificial intelligence (AI) to track diseases tha
Disc Medicine has charted a route to market for bitopertin, its drug candidate for rare disease erythropoietic protoporphyria (EPP), after meeting with the FDA.