Down syndrome is a rare chromosomal disorder that occurs when a person has a partial or total extra copy of chromosome 21. It is also known trisomy 21, characterized by lifelong mental retardation, muscular hypotonia, and poor muscle tone, often associated with a characteristic distinct facial appearance and other problems such as developmental delays, flattened facial features, short neck, small head, protruding tongue, and anomalies such as cardiac, gastrointestinal, or endocrine defects. The cause of the disease is still unknown. Development of the children with Down syndrome varies in severity; therefore, development problems range from moderate to serious. It is not possible to predict the future development of children with this syndrome. Children with Down syndrome may also have small, upturned, almond-shaped eyes, small facial features, large tongue, and broad, short hands with a single crease on the palm, poor muscle tone, relatively short fingers and excessive flexibility. According to the Centers for Disease Control and Prevention, in the U.S. nearly 1 in every 1,000 children and teenagers (0 to 19 years old) had Down syndrome in 2002. Around 83,000 children and teenagers were living with this syndrome in the U.S in 2012.
Increase in research & development, introduction of novel disease therapies, rise in demand for effective therapies among patients, and increase in prevalence of the disease are projected to fuel the global Down syndrome market during the forecast period. According to the European Down syndrome Association (EDSA), Down syndrome occurs at the rate of about 1 per 1000 births. Presently, the life expectancy of a person with this syndrome is around 60 to 65 years, with some living even beyond this age in Europe. In the U.S the average medical care costs were 12 times higher for Down syndrome children aged between 0 and 4 years as compared to children without Down syndrome of the same age with private insurance.
The global Down syndrome market can be segmented based on disease type, treatment, and region. In terms of disease type, the Down syndrome market can be categorized into complete trisomy 21, translocation trisomy 21, and mosaic trisomy 21. Based on treatment, the Down syndrome market can be divided into occupational therapies, speech therapies, physical therapies, and others. No specific treatment is known that can cure this syndrome; however, seizures can be reduced with anticonvulsant drugs, and other medical problems can be reduced and controlled by certain therapies. As of March 2018, the Down syndrome treatment drugs pipeline comprises 14 drug candidates, which are in different stages of development. These pipelines include gene therapy, small molecules, vaccines, and others drug candidates that are developed as recombinant peptides and proteins. Most drug candidates for the treatment of this syndrome are in the clinical stage of development. This is expected to lead to more drug approvals for the treatment of this syndrome. For instance, NN 351 exhibited positive results in the pre-clinical trial by reducing the overall hyperactivity and increasing learning and memory abilities in transgenic mice with Down syndrome.
In terms of region, the global Down syndrome market can be categorized into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America accounted for a major share of the global Down syndrome market. According to a Research Office Legislative Council Secretariat report, there are over 30 million people suffering from rare diseases in the U.S. Strong focus on R&D and government aid to promote R&D are projected to propel the Down syndrome market. Europe was the trailing region in terms of revenues in the rare disease treatment drugs sector in the world.
Players operating in the global Down syndrome market include Eisai Co Ltd, F. Hoffmann-La Roche Ltd, NeuroNascent, Inc., AC Immune, Aelis Farma SAS, QR Pharma, Inc., and ManRos Therapeutics.
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