Neuromuscular diseases

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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

News

Scholar Rock preps filings after SMA antibody clears trial

Shares in Scholar Rock have gone into overdrive after the biotech reported positive phase 3 results with apitegromab in patients with spinal muscular atrophy (SMA), setting up regulatory fi

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Surfing on new Duchenne data, Wave charges $175m offering

Phase 2 results with Wave Life Sciences' Duchenne muscular dystrophy candidate WVE-N531 pushed the company's share price to its highest level in a year and could lead to discussions with re

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Sanofi's tolebrutinib cuts MS progression by 31% in trial

Sanofi has bolstered the case for its oral BTK inhibitor tolebrutinib as a treatment for multiple sclerosis (MS), due to be filed for approval in the latter half of this year, with new data

News

Roche trumpets new fenebrutinib data in relapsing MS

Data from a phase 2 trial of Roche's oral BTK inhibitor fenebrutinib have shown "near-complete suppression" of disease activity and disability progression in relapsing multiple sclerosis (M

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Two of three trials fail, but Sanofi will still file MS drug

Sanofi has said it plans to start discussions about filing its oral BTK inhibitor tolebrutinib for multiple sclerosis, even though the drug only achieved its primary objective in one of thr

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