Huntington’s disease is a hereditary neurodegenerative disease that results in motor, cognitive and psychiatric disability, primarily due to the destruction of neurons in the brain. The National Institutes of Health estimates that 30,000 people in the United States have Huntington’s disease. One of the first symptoms of this disease is chorea, which occurs in 90% of patients. In approximately 70% of these patients, chorea is moderate to severe and can result in difficulty walking, speaking, swallowing or performing simple everyday tasks.
Presentation of SD-809 for the Treatment of Chorea Associated with Huntington's DiseaseJune 22, 2013 Huntington's Disease Society of America Author: David Stamler, M.D. Chief Medical Officer, Auspex Pharmaceuticals, Inc. Download Presentation »
Tourette syndrome is a hyperkinetic movement disorder manifested by motor and vocal tics, which are often accompanied by behavioral disorders such as attention-deficit hyperactivity disorder, or ADHD, and obsessive-compulsive disorder, or OCD. Tics can be simple such as blinking, eye rolling, nose twitching, head nodding and mouth pouting, or more complex such as touching, squatting, jumping or hopping. Tics can result in significant long-term social, legal and developmental consequences for patients, as well as physical disability including pain and secondary neurological deficits. In the United States, an estimated 100,000 to 350,000 children have tics associated with Tourette syndrome. According to the U.S. Centers for Disease Control and Prevention, 27% of these children have moderate to severe forms of Tourette syndrome. The mean age of onset is at four to six years, with peak severity around 12 years of age, with 13% to 22% of affected children continuing to take medications for tics as adults. There have been no new drugs introduced for treating tics associated with Tourette syndrome in 30 years. These treatments carry, among other adverse events, the risk of causing permanent neurologic deficits, such as tardive dyskinesia.
Tardive dyskinesia is a hyperkinetic movement disorder that is induced by dopamine receptor blocking agents, such as neuroleptics, which are used for treating psychiatric conditions, including schizophrenia and bipolar disease, as well as by certain drugs, such as metoclopramide, which are used for treating various gastrointestinal disorders. Neuroleptics are estimated to be used by approximately four million Americans according to federal government data from the Medical Expenditure Panel Survey. Tardive dyskinesia typically manifests as rapid and repetitive movements involving the tongue, lips and jaw that may involve puffing of cheeks, protruding of the tongue, lip smacking, puckering, pursing and chewing. In the United States, an estimated 500,000 patients have tardive dyskinesia. There are no FDA-approved treatments for the condition.