Neuromuscular diseases

News

FDA advisors vote no to Capricor's DMD therapy

FDA advisors voted that Capricor has not shown efficacy for deramiocel in Duchenne muscular dystrophy, due to altered endpoints in its main trial.

News

As patents expire, fampridine is cleared for NHS use in MS

Several years after other UK nations, people living with multiple sclerosis in England are finally able to get NHS treatment with fampridine.

News

Servier wagers $2.65bn on Edgewise muscular dystrophy drug

Servier has signed the largest licensing deal in its history, paying $1.55bn upfront for Edgewise's muscular dystrophy business headed by sevasemten.

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.

News

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.

News

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.

FDA advisors vote no to Capricor's DMD therapy

As patents expire, fampridine is cleared for NHS use in MS

Servier wagers $2.65bn on Edgewise muscular dystrophy drug

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

TransThera's resistant biliary cancer drug cleared in China

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

Neuromuscular diseases

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