Research over the last 10 years has unveiled numerous disease mechanisms in amyotrophic lateral sclerosis (ALS), most of which act in motor neurons. These new insights into ALS biology are allowing drug developers to shift from focusing on downstream targets and pathways, such as inflammation or muscle weakness, to upstream mechanisms more likely to drive the disease.

Therapies currently under development may target one of several processes linked to ALS, including:

1 Inflammation of the cell body

2 Aggregation of proteins in the cell cytoplasm

3 Defects in nuclear pore complexes

4 Dysregulation of mRNA processing

5 Dysregulation of microRNAs

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