OTHER MEDICAL USES
for Sodium Phenylbutyrate

African swine fever is a highly contagious and deadly viral disease affecting both domestic and wild pigs of all ages. ASF is not a threat to human health and cannot be transmitted from pigs to humans. It is not a food safety issue. ASF is found in countries around the world, particularly in sub-Saharan Africa.

Alzheimer’s disease is an irreversible, progressive brain disorder that slowly destroys memory and thinking skills and, eventually, the ability to carry out the simplest tasks. In most people with the disease – those with the late-onset type – symptoms first appear in their mid-60s.  Early-onset Alzheimer’s occurs between a person’s 30s and mid-60s and is very rare. Alzheimer’s disease is the most common cause of dementia among older adults.

Bovine mastitis is one of the most significant diseases of dairy cattle. Mastitis is an infectious disease condition resulting in an inflammatory reaction in the mammary gland of the cow. It may be accompanied by signs of inflammation in the mammary gland including swelling, redness, and pain.

Cerebral ischemia is a condition in which a blockage in an artery restricts the delivery of oxygen-rich blood to the brain, resulting in damage to brain tissue. Cerebral ischemia is sometimes called brain ischemia or cerebrovascular ischemia.

Cystic fibrosis is a progressive, genetic disease that causes persistent lung infections and limits the ability to breathe. In people with CF, mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene cause the CFTR protein to become dysfunctional. When the protein is not working correctly, it’s unable to help move chloride – a component of salt – to the cell surface. Without the chloride to attract water to the cell surface, the mucus in various organs becomes thick and sticky.

In the lungs, the mucus clogs the airways and traps germs like bacteria, leading to infections, inflammation, respiratory failure, and other complications. For this reason, minimizing contact with germs is a top concern for people with CF.

In the pancreas, the buildup of mucus prevents the release of digestive enzymes that help the body absorb food and key nutrients, resulting in malnutrition and poor growth. In the liver, the thick mucus can block the bile duct, causing liver disease. In men, CF can affect their ability to have children.

Endoplasmic reticulum (ER) stress occurs when the capacity of the ER to fold proteins becomes saturated. ER stress may be caused by factors that impair protein glycosylation or disulfide bond formation, or by overexpression of or mutations in proteins entering the secretory pathway.

Epilepsy may occur as a result of a genetic disorder or an acquired brain injury, such as a trauma or stroke.  During a seizure, a person experiences abnormal behavior, symptoms, and sensations, sometimes including loss of consciousness. There are few symptoms between seizures.  Epilepsy is usually treated by medications and in some cases by surgery, devices, or dietary changes.

Huntington’s disease is a progressive brain disorder that causes uncontrolled movements, emotional problems, and loss of cognition. Adult-onset Huntington’s disease, the most common form of this disorder, usually appears in a person’s thirties or forties.

Early signs and symptoms can include irritability, depression, small involuntary movements, poor coordination, and trouble learning new information or making decisions. Many people with Huntington’s disease develop involuntary jerking or twitching movements known as chorea. As the disease progresses, these movements become more pronounced.

Affected individuals may have trouble walking, speaking, and swallowing. People with this disorder also experience changes in personality and a decline in thinking and reasoning abilities. Individuals with the adult-onset form of Huntington’s disease usually live about 15 to 20 years after signs and symptoms begin.

A less common form of Huntington’s disease, the juvenile form, begins in childhood or adolescence. It also involves movement problems and mental and emotional changes.

Additional signs of the juvenile form include slow movements, clumsiness, frequent falling, rigidity, slurred speech, and drooling. School performance declines as thinking and reasoning abilities become impaired. Seizures occur in 30% to 50% of children with this condition. Juvenile Huntington’s disease tends to progress more quickly than the adult-onset form. Affected individuals usually live 10 to 15 years after signs and symptoms appear.

Spinal cord ischemia is a relatively uncommon form of spinal cord injury. The true prevalence of cord ischemia is not known, but it is suggested that fewer than 2% of central neurovascular events affect the cord and fewer than 8% of all acute myelopathies have an ischemic component.

The infrequent nature of spinal cord ischemia is due to the multiple collateral sources of blood supply. Rostrally, branches from the vertebral arteries come together in the midline to form the anterior spinal artery. Caudally, blood to the anterior cord is provided by radicular arteries which arise from the aorta at multiple levels and anastomose with dural branches posteriorly.

Kidney disease means kidneys are damaged and can’t filter blood the way they should. Someone is at greater risk for kidney disease if they have diabetes or high blood pressure. Treatments include kidney transplant or dialysis.

Maple syrup urine disease is an inherited disorder in which the body is unable to process amino acids properly. The condition gets its name from the distinctive sweet odor of affected infants’ urine. It is also characterized by poor feeding, vomiting, lack of energy, abnormal movements, and delayed development. If untreated, maple syrup urine disease can lead to seizures, coma, and death.

Maple syrup urine disease is often classified by its pattern of signs and symptoms. The most common and severe form of the disease is the classic type, which becomes apparent soon after birth. Variant forms of the disorder become apparent later in infancy or childhood and are typically milder, but they still lead to delayed development and other health problems if not treated.

In multiple sclerosis, resulting nerve damage disrupts communication between the brain and the body. Multiple sclerosis causes many different symptoms, including vision loss, pain, fatigue, and impaired coordination. The symptoms, severity, and duration can vary from person to person.

Some people may be symptom-free most of their lives, while others can have severe chronic symptoms that never go away. Physical therapy and medications that suppress the immune system can help with symptoms and slow disease progression.

Parkinson’s disease is a brain disorder that leads to shaking, stiffness, and difficulty with walking, balance, and coordination. Parkinson’s symptoms usually begin gradually and get worse over time. As the disease progresses, people may have difficulty walking and talking.

Pyruvate dehydrogenase complex (PDC) deficiency is a type of metabolic disease. This means that the body is not able to efficiently break down nutrients in food to be used for energy. Symptoms of PDC deficiency include signs of metabolic dysfunction such as extreme tiredness (lethargy), poor feeding, and rapid breathing (tachypnea).

Other symptoms may include signs of neurological dysfunction such as developmental delay, periods of uncontrolled movements (ataxia), low muscle tone (hypotonia), abnormal eye movements, and seizures. Symptoms usually begin in infancy, but signs can first appear at birth or later in childhood. Symptoms may be especially apparent during times of illness, stress, or after meals with high amounts of carbohydrates.

Schizophrenia is characterized by thoughts or experiences that seem out of touch with reality, disorganized speech or behavior, and decreased participation in daily activities. Difficulty with concentration and memory may also be present. Treatment is usually lifelong and often involves a combination of medications, psychotherapy, and coordinated specialty care services.

Sickle cell disease is an inherited group of disorders where red blood cells contort into a sickle shape. The cells die early, leaving a shortage of healthy red blood cells (sickle cell anemia), and can block blood flow causing pain (sickle cell crisis). Infections, pain, and fatigue are symptoms of sickle cell disease. Treatments include medications, blood transfusions, and rarely a bone marrow transplant.

Thalassemia is an inherited blood disorder characterized by less oxygen-carrying protein (hemoglobin) and fewer red blood cells in the body than normal. Symptoms include fatigue, weakness, paleness, and slow growth. Mild forms may not need treatment. Severe forms may require blood transfusions or a donor stem-cell transplant.

Spinal muscular atrophy (SMA) is a progressive, rare genetic disease that is caused by the survival motor neuron 1 (SMN1) gene that is missing or not working properly.

The liver in a person with urea cycle disorder is missing an enzyme necessary to convert nitrogen into urea. As a result, ammonia, a highly toxic substance, builds up in the bloodstream and is not removed from the body. Untreated, the high amounts of ammonia can cause brain damage, coma, and eventually death.