Neuromuscular diseases

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Boy in a wheelchair looking out to the forest

Regenxbio, Solid report data with Duchenne gene therapies

Regenxbio and Solid Bio say their gene therapies for Duchenne muscular dystrophy show no sign so far of the liver issues seen with Sarepta's Elevidys.

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PepGen slumps as FDA places hold on lead drug

Shares in PepGen fell sharply after the FDA placed a partial clinical hold on its lead drug for rare neuromuscular disease DM1.

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PTC pulls file for Duchenne therapy on FDA feedback

PTC Therapeutics has abandoned its latest attempt to secure FDA approval for Duchenne muscular dystrophy treatment Translarna.

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Boy in wheelchair, only lower half of his body visible

Novartis' SMA gene therapy cleared for wider use in US

A new FDA approval means Novartis now has a gene therapy option for almost everyone with spinal muscular atrophy (SMA), regardless of their age.

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Sarepta shares slide as DMD treatment woes pile up

The ESSENCE trial of Sarepta's Vyondys 53 and Amondys 45 failed to hit its objective, placing their FDA accelerated approvals at risk.

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Novartis makes $12bn wager on RNA drugs with Avidity buy

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

Regenxbio, Solid report data with Duchenne gene therapies

PepGen slumps as FDA places hold on lead drug

PTC pulls file for Duchenne therapy on FDA feedback

Novartis' SMA gene therapy cleared for wider use in US

Sarepta shares slide as DMD treatment woes pile up

Novartis makes $12bn wager on RNA drugs with Avidity buy

Chiesi signs $1.9bn deal to buy KalVista and its HAE drug

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Neuromuscular diseases

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