Amyotrophic Lateral Sclerosis(ALS)
Amyotrophic Lateral Sclerosis (ALS), more commonly known as Lou Gehrig’s disease, is a progressive and fatal neuromuscular disease characterized by muscle atrophy and muscle loss due to the destruction of motor neurons. There is no known cause nor identified cure for ALS.
The disease is diverse in its presentation, cause, and progression, but, in general, ALS presents clinically as asymmetric muscle weakness, wasting, spasticity, weight loss, dysphagia, and paralysis; in 10% to 15% of patients, cognitive impairment may arise before or following that of motor neuron dysfunction. Estimates of the incidence of ALS worldwide range from 1 to 3 cases per 100,000 people annually. The ALS Association, the Centers for Disease Control and Prevention, and the National Institute of Neurological Disorders and Stroke estimate that as many as 30,000 Americans may have the disease at any given time, with approximately 5,600 newly diagnosed patients annually.
When Engensis (VM202) is injected along the peripheral nerves and their branches, the internal production of HGF may protect nerves and promote the growth of neurons while also ameliorating atrophic conditions. The US FDA granted orphan drug and fast track designation for Engensis (VM202) in 2016.