Experimental motor neurone disease therapy shows promise – European Pharmaceutical Review

reduced expression of the SOD1 gene known to cause amyotrophic lateral sclerosis (ALS) and slowed clinical decline in the Phase I/II trial.

Tofersen, an experimental therapy developed to address the genetic cause of a amyotrophic lateral sclerosis (ALS), rare form of motor neurone disease (MND) was shown to be safe and well tolerated in a Phase I/II trial.

Approximately 5,000 people in the UK have MND, a disorder that causes a gradual degeneration in the connectivity between neurons of the central nervous system and the muscles in the rest of the body, leading them to weaken, stiffen and eventually waste, affecting a patient’s ability to walk, talk, use their arms and hands, eat and breathe.

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