Neuromuscular diseases

News

Angelini CEO Sergio Marullo di Condojanni

Angelini seeks US beachhead with $4.1bn Catalyst buy

Angelini has agreed to buy Catalyst Pharma for up to $4.1bn, its first acquisition since 2021, which would mark its entry into the US market.

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NICE backs UCB drug as first uncontrolled gMG therapy

UCB's Rystiggo can be used by the NHS as a backup treatment for people with the rare autoimmune and neuromuscular disease gMG, says NICE.

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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.

News

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.

Angelini seeks US beachhead with $4.1bn Catalyst buy

NICE backs UCB drug as first uncontrolled gMG therapy

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

OpenBind unveils its first AI model for drug discovery

Discover a new era of healthcare panel engagement: Grounded ...

Neuromuscular diseases

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