Neurology Central

Investigational therapy for ALS now in clinical trials

Researchers from the Washington University School of Medicine (MO, USA) have investigated a new therapy for inherited amyotrophic lateral sclerosis (ALS), which has extended survival and reversed fatal neuromuscular damage in rodents, leading to Phase I/II clinical trials.

ALS, also known as Lou Gehrig’s disease, affects approximately 20,000 people in the USA, and around 2% of these cases are caused by the inherited mutation and consequential upregulation of SOD1. The disease destroys nerve cells, preventing walking, eating and breathing, and survival for more than 3 years after diagnosis is rare. There are currently only two US FDA-approved drugs available to patients and these only extend lifespan by a short while.

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