Neurological Disorders Research

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, is a type of motor neuron disease. It affects the nerve cells in the brain and spinal cord that control voluntary muscle movement and breathing. In most cases it does not affect intelligence, thinking, sight or hearing unless associated, although more rarely, with a form of dementia such as frontotemporal dementia (FTD).
Limb weakness, slurred speech, or problems swallowing are some of the first symptoms that can develop. Over time, symptoms progress and cause trouble with breathing, swallowing and paralysis.
The condition most commonly affects people between the ages of 55 and 75. The causes of ALS are not yet fully understood, but researchers think it may be due to genes and environment. There is no cure for ALS available yet, but clinical research studies are looking at potential treatments for people living with ALS.

Frontotemporal dementia (FTD) is also known as frontotemporal degeneration. It is caused by nerve cell loss in the front and sides of the brain (the frontal and temporal lobes). FTD can cause changes in behavior, personality and can make speaking and understanding others difficult. Some people with FTD may also develop movement disorders. Symptoms usually start gradually and get worse over time.
FTD is the second most common type of dementia in people under the age of 65, after Alzheimer’s. Treatments are available to help with symptoms, but there is no cure available yet.
Some clinical research studies for FTD are using gene therapy. To learn more about this type of research visit our genetic disorders page.

Parkinson’s Disease is also known as Parkinson’s or Parkinson’s disorder. It is a brain disorder that causes movement issues, including shaking or ‘tremor’, stiffness, and difficulty with balance and coordination.
Symptoms usually start gradually and get worse over time. It happens when nerve cells in the brain that control movement are damaged and produce less dopamine. Dopamine is a brain chemical that controls movement and allows you to feel pleasure and motivation.
Treatments are available to help with symptoms, but there is no cure available yet.
Some clinical research studies for Parkinson’s disease are using gene therapy. You can learn more about gene therapy research for Parkinson’s disease on the American Society of Gene & Cell Therapy website. You can also visit our genetic disorders page.

Gaucher disease is a rare genetic condition that is passed down from parents to children. People with Gaucher disease are missing an enzyme that normally breaks down lipids. Lipids are fatty substances found in the body. Lipids start to build up in organs including the spleen and liver. This can cause many different symptoms. The spleen and liver can get very large and stop working normally. The disease can also cause anemia and affect the lungs, eyes, and bones. Some types of this disease can also affect the brain.
Treatments are available to help with symptoms, but there is no cure available yet. Some clinical research studies for Gaucher disease are using gene therapy. These studies are looking at two different types of Gaucher disease:

• type 1 Gaucher disease which is seen in children and adults and can be mild or severe.
• type 2 Gaucher disease which is a severe form of the disease and affects babies.

To learn more about this type of research visit our genetic disorders page.