Neuromuscular diseases

News

A new study suggests long-read 'nanopore' sequencing could transform the diagnosis of inherited diseases and unlock new insights for drug discovery.

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Scholar Rock has won its first product approval, on its second attempt, as the FDA gives a green light to its spinal muscular atrophy drug Isembyld.

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Novartis has suffered another pipeline blow, this time involving its highly anticipated neuromuscular disease therapy del-desiran.

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

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

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