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Fighting Neurological Disorders

Biopharmaceutical companies are developing more than 400 new medicines to prevent and treat neurological disorders.

Neurological disorders—such as epilepsy, multiple sclerosis and Parkinson’s disease—inflict great pain and suffering on patients and their families. 

In recent years, scientists have learned more about how the nervous system works at the molecular and genetic levels, helping to develop more effective treatments for neurological disorders. However, many disorders still do not have an approved treatment or are in need of newer, more effective treatments.

Biopharmaceutical research companies are in the process of developing 420 medicines to prevent and treat a number of neurological disorders. The medicines in development are either in human clinical trials or under review at the Food and Drug Administration (FDA).

30,000 Americans with Huntington's

It is estimated that 30,000 Americans have Huntington's and another 200,000 are at-risk of inheriting the disease. Recently approved treatments are helping patients manage symptoms and improve their quality of life by modifying chemical pathways in the brain to reduce involuntary movements (chorea). Lessening the burden of chorea allows HD patients to take part in normal daily activities.

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Spotlight: Alzheimer's Disease

Alzheimer’s is the sixth-leading cause of death in the United States, and its impact on patients and the health care system is growing. Existing medicines treat the symptoms of the disease but cannot slow, prevent or reverse the progressive deterioration and dementia it causes. The development of disease modifying treatments that delay the onset of Alzheimer’s could reduce Medicare and Medicaid spending on patients with Alzheimer’s by more than $218 billion annually by the year 2050. Researchers continue to unravel the mysteries of the disease and are studying many new treatments in this area. Recent research has focused on the plaques and tangles that form in the brains of Alzheimer’s patients and are thought to contribute to the death of nerve cells.

New Meds in Development- Neurological Disorders


There are more than 600 neurological disorders that strike millions of Americans each year. This includes, Alzheimer’s, brain tumors, epilepsy, Huntington’s Disease, Muscular Dystrophy, Multiple Sclerosis, Parkinson’s, and stroke, amongst others.

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50 Million

Estimated Number of Americans Affected by Neurological Disorders

Although some are well known, such as epilepsy, multiple sclerosis and Parkinson’s disease, many are rare disorders affecting just a small number of patients. Neurological disorders represent eight percent of the global health burden, with stroke, migraine, and epilepsy being the top contributors.

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Medicines in the Pipeline

America’s biopharmaceutical research companies are currently developing 420 new medicines to prevent and treat neurological disorders. These medicines in development include treatments that explore new pathways and scientific approaches to treat these complex disorders. Our growing understanding of the underlying mechanisms of neurological disorders have helped advance multiple sclerosis (MS) treatment from painful site injections to a broad range of convenient options that reduce relapses, prevent disease progression, and improve quality of life. Read our report on medicines in development for MS and other challenging autoimmune diseases, and learn about forthcoming treatments for other neurological diseases here:

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Related Medicines

  • Migraine Prevention

    A monoclonal antibody in development for the prevention of migraine binds to and inhibits the activity of calcitonin generelated peptide (CGRP). CGRP is expressed in the nervous system, where it plays a role in controlling the widening of blood vessels and the transmission of nociceptive pain (pain arising from nerve cells) information. Research suggests that CGRP pathways may be involved in the development of migraines. By inhibiting CGRP activity, anti-CGRP antibodies are thought to help inhibit the transmission of pain signals associated with migraines.

  • Epilepsy

    A medicine, already approved for use by adults, is in clinical trials for pediatric partial onset seizures. Seizures begin in the brain, which is made up of millions of nerve cells which communicate with each other by sending electrical signals. In people with epilepsy, overexcited nerve cells send too many electrical signals that can then cause seizures. The medicine works by reducing the number of “extra” electrical signals that are sent out. The medicine is also being studied in pediatric and adult populations for primary generalized tonic-clonic seizures.

  • Huntington's

    A medicine in development for Huntington’s disease targets the PDE10A enzyme, which is present in the neurons most damaged in Huntington’s. Huntington’s disease is an inherited disorder caused by the programmed degeneration of brain cells, called neurons, in certain areas of the brain. This degeneration can lead to uncontrolled movements, loss of intellectual faculties, and emotional disturbances. Inhibition of the PDE10A enzyme has been shown to improve cell function and potentially provide neuroprotective effects.

  • ALS

    A monoclonal antibody in development for amyotrophic lateral sclerosis (ALS) is an inhibitor of the protein Nogo-A. Research suggests that Nogo A may be involved in the pathology of ALS. Motor neurons signal the body’s muscles to move. In ALS, the junctions that transmit signals from the motor neurons to the muscles begin to degenerate, leading to muscle weakness, paralysis, and respiratory failure. Inhibiting Nogo-A protein may help keep the motor neurons and muscle fibers connected in hopes that it will lead to a slowing or stopping of disease progression.



View more information on medicines in development for neurological diseases: