Sodium Phenylbutyrate
ALS RESEARCH

Sodium phenylbutyrate ALS research studies are contributing to worldwide knowledge about ALS and have shown promising results including prolonged survival, regulated expression of anti-apoptotic genes, and a reduction in neuron loss.

AMYOTROPHIC LATERAL SCLEROSIS (ALS)

Amyotrophic lateral sclerosis (ALS) is a group of rare neurological diseases that mainly involve the nerve cells (neurons) responsible for controlling voluntary muscle movement. Voluntary muscles produce movements like chewing, walking, and talking. The disease is progressive, meaning the symptoms get worse over time. Currently, there is no cure for ALS and no effective treatment to halt or reverse the progression of the disease.

ALS belongs to a wider group of disorders known as motor neuron diseases, which are caused by gradual deterioration and death of motor neurons. Motor neurons are nerve cells that extend from the brain to the spinal cord and muscles throughout the body. These motor neurons initiate and provide vital communication links between the brain and the voluntary muscles.

Messages from motor neurons in the brain, called upper motor neurons, are transmitted to motor neurons in the spinal cord and to motor nuclei of the brain, called lower motor neurons, and from the spinal cord and motor nuclei of the brain to a particular muscle or muscles.
In ALS, both the upper motor neurons and the lower motor neurons degenerate or die and stop sending messages to the muscles. Unable to function, the muscles gradually weaken, start to twitch in spasms called fasciculations, and waste away. Eventually, the brain loses its ability to initiate and control voluntary movements.

Early symptoms of ALS usually include muscle weakness or stiffness. Gradually, all muscles under voluntary control are affected, and individuals lose their strength and the ability to speak, eat, move, and even breathe. Most people with ALS die from respiratory failure, usually within 3 to 5 years from when the symptoms first appear. However, about 10% of people with ALS survive for 10 or more years.