Neuromuscular diseases

News

Novartis is poised to make one of its largest takeover deals in its history after agreeing to buy RNA specialist Avidity Biosciences for $12bn.

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It looks like PTC will have to carry out another trial of its drug for Friedreich ataxia before the FDA will consider approval.

News

Novartis has made an offer to acquire RNA therapeutic specialist Avidity Biosciences, according to an unconfirmed report.

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Roche has revealed good results from a phase 2 trial of its oral BTK drug fenebrutinib in relapsing multiple sclerosis as it waits for phase 3 data.

News

Sanofi's oral BTK inhibitor tolebrutinib is heading towards an FDA decision in September as a potential treatment for multiple sclerosis.

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Phase 3 results with an intrathecal version of Novartis spinal muscular atrophy (SMA) gene therapy could make older children eligible for treatment.