Neuromuscular diseases

News

PTC stung by FDA decision not to approve rare disease drug

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

News

Rumour mill says Novartis may want to buy Avidity

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

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Roche builds case for MS drug with two-year efficacy data

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.

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FDA starts review of Sanofi's MS hope tolebrutinib

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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New data could widen use of Novartis' SMA therapy Zolgensma

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

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

PTC stung by FDA decision not to approve rare disease drug

Rumour mill says Novartis may want to buy Avidity

Roche builds case for MS drug with two-year efficacy data

FDA starts review of Sanofi's MS hope tolebrutinib

New data could widen use of Novartis' SMA therapy Zolgensma

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

FDA points to liver injury with Amgen's Tavneos

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Neuromuscular diseases

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