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A Rundown Of Dravet Syndrome Drugs In The Pipeline

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Rare commodities are worth more than good is a Chinese adage. And more so when it is in the field of medicine.

Rare diseases, also called orphan diseases, as the name implies, affect very small numbers of patients. Estimates by the National Human Genome research Institute pitch the number of rare diseases at over 6,800. Childhood cancers, cystic fibrosis, Huntington's disease, muscular dystrophy, cystinosis, Dravet syndrome and retinitis pigmentosa are some examples of rare diseases.

By U.S. standards, if a disease affects less than 200,000 people then it is classified as a rare disease.

In this article, we will give an overview of one of the rare diseases, Dravet syndrome, and the companies developing drugs for this indication.

Dravet syndrome, named after French psychiatrist and epileptologist Charlotte Dravet who first identified this condition, is a rare distinctive epileptic syndrome that occurs typically during the first year of life in an otherwise healthy and developmentally normal infant.

Individuals with Dravet syndrome face a higher incidence of *status epilepticus and Sudden Unexplained Death in Epilepsy, or SUDEP. (*Status epilepticus is when a seizure lasts longer than 5 minutes or when seizures occur close together and the person doesn't recover between seizures).

The most common gene mutation linked to Dravet syndrome, formerly known as Severe Myoclonic Epilepsy in Infancy (SMEI), is in a gene called SCN1A. When this gene isn't working properly, sodium channels in the brain (which help brain cells function) do not work correctly. (Source: Epilepsy Foundation).

It is estimated that there are between 16,000 and 29,000 patients living with Dravet syndrome in the United States and Europe. There is no FDA-approved drug yet for this disorder. The standard of care for the treatment of Dravet syndrome involves a combination of anticonvulsant drugs. The mortality rate in Dravet syndrome is estimated to range from 10-15%.

Here's a list of some of the companies developing drugs for Dravet syndrome, and their current pipeline status.

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