ataxia

News

PTC slides on failed Friedreich ataxia study

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

News

Healx deploys its AI in hunt for Friedreich's ataxia therapi...

UK artificial intelligence specialist Healx has begun a partnership with two medical charities that aims to uncover new therapies for the neurodegenerative disease Friedreich's ataxia (FA).

News

Gene therapy firm Kate Tx snapped up by Novartis for $1.1bn

Novartis buys gene therapy specialist Kate Therapeutics for $1.1 billion, adding an early-stage rival to Sarepta's muscular dystrophy therapy Elevidys

Sales & Marketing

Coming soon: The Life Sciences Industry Report 2025 – insigh...

Prepare for 2025 with pharmaphorum’s Life Sciences Report, featuring expert insights on AI in drug development, gene therapy, and top industry trends

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