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13 Jul 2022

New treatment for motor neurone disease shows promise in early trials

A new drug targeting the genetic cause of a rare form of motor neurone disease (MND) has shown encouraging results in early research supported by the National Institute for Health and Care Reseaerch (NIHR).

The phase 1-2 study, published in the New England Journal of Medicine, has shown that the new investigational drug tofersen is safe and well tolerated in people with a genetic form of MND.

Read the NIHR article.

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