Amyotrophic Lateral Sclerosis (ALS)

Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s Disease, or Motor Neuron Disease is a rare, progressive neurodegenerative disease that is ultimately fatal. In ALS, the motor neurons in the brain and spinal cord die, resulting in weakness, muscle atrophy, paralysis and frequently, cognitive impairment, before resulting in death from respiratory failure. Each year, more than 5,000 people in the U.S. are diagnosed with ALS and an estimated 20,000 are living with the disease.

Our efforts to advance the treatment of Amyotrophic Lateral Sclerosis

We are conducting pioneering work in the search for genetic treatments that target the dysfunctional brain immune system to slow the progression of neurodegenerative diseases like ALS.

Researchers have come to understand that ALS and frontotemporal dementia are part of a broad neurodegenerative continuum and can share disease pathology and presenting characteristics, including behavioral, cognitive or language deficits. Decreased progranulin levels are associated with the abnormal accumulation of the TAR DNA-binding protein 43 (TDP-43). Excess aggregation of TDP-43 is thought to lead to neuronal death. We are developing AL001, our lead therapeutic designed to elevate progranulin levels, for ALS based on evidence that increasing progranulin levels may be protective, and even reverse, TDP-43 pathology.

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