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Target ALS

A New York based ALS research organization that's leading the fight for a cure.

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Glossary

Amyotrophic lateral sclerosis, more commonly known as ALS, is a progressive neurodegenerative disease in the central nervous system that impacts the brain and spinal cord, resulting in a loss of muscle control.

It is colloquially referred to as Lou Gehrig’s Disease, after the famous baseball player who battled it in the late 1930s, and does not yet have a cure. To better understand the language of ALS, the ALS Glossary below provides definitions for research terms commonly used by our Innovation Ecosystem and the extended ALS community.

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pALS (person with ALS)

A common term in the ALS community that refers to someone diagnosed with ALS.

palsy

Paralysis that is often accompanied by involuntary tremors.

Parkinson's disease

A neurodegenerative disease caused by the loss of dopamine-producing brain cells resulting in difficulty controlling movement.

Pathobiology

Although sometimes used interchangeably with pathology, pathobiology places greater emphasis on the biological aspects rather than the medical aspects.

Pathophysiology

The functional changes in the body associated with or resulting from disease or injury. It also refers to the study of such changes. It is a biological science, whereas pathology is a medical discipline.

pharmacodynamic biomarker

A type of biomarker that helps to evaluate whether a treatment is working.

pharmacology

The study of drugs, their properties and effects.

phenotype

An individual's observable traits (e.g., height, eye color, blood type).

plasmid

Small circular pieces of DNA that can be replicated when inserted in cells. Plasmids can be delivered to cells in vectors in recombinant DNA research.

poly (GA)

A dipeptide repeat protein (DPR) that accumulates in ALS as a result from mutations in the C9orf72 gene. DPRs have the potential to be used as biomarkers. Poly (GA) is the most abundant DPR in ALS and FTD patients with mutations in C9orf72, and contributes to TDP-43 abnormalities and neuron loss

poly (GP)

A potential biomarker for ALS and FTD, poly (GP) is a dipeptide repeat protein (DPR) resulting from mutations in the C9orf72 gene. Increased levels of poly (GP) have been found in the spinal fluid of ALS and FTD patients with a C9orf72 mutation

poly (GR)

A dipeptide repeat protein (DPR) that accumulates in ALS as a result from mutations in the C9orf72 gene. DPRs have the potential to be used as biomarkers. Poly (GR) may be the most toxic DPR to cells, and animal research suggests suppressing the production of Poly (GR) prevents its aggregation and ensuing neurodegeneration

primary lateral sclerosis

A motor neuron disease closely related to ALS. It affects only the upper motor neurons and progresses more slowly than ALS.

prognostic biomarker

A type of biomarker that provides information on disease progression and/or outcome.

progressive bulbar palsy

A motor neuron disease that involves the brain stem—the bulb-shaped region containing lower motor neurons involved in swallowing, speaking, chewing and other functions. Initially, patients with progressive bulbar palsy only have muscle weakness that affects speech and swallowing. However, this condition often progresses to ALS

proteins

Proteins are the building blocks in all cells in all organisms. To produce them, DNA from genes is first transcribed into RNA, then RNA is translated into proteins.

pseudobulbar palsy

A motor neuron disease closely related to ALS that primarily affects the ability to speak, swallow and chew. These symptoms resemble those of bulbar palsy, but this condition is also characterized by spontaneous or unmotivated crying and laughing

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