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DiGeorge syndrome is a group of signs and symptoms associated with developmental defects in the pharynx system.
of DGS are cardiac conical artery dry malformation, thymus dysplptia, and hypocalcemia (caused by thyroid dysplia).
chromosome 22q11.2 is present in most DGS patients and other similar syndromes such as cardiac syndrome (also known as Shprintzen syndrome).
these diseases fall into the category of chromosomal 22q11.2 deficiency syndrome.
pharmaceutical company Zynerba Pharmaceuticals announced today that the U.S. Food and Drug Administration (FDA) has awarded Cannabidiol the title of orphan drug for the treatment of 22q11.2 deficiency syndrome.
cannabidiol is the main non-psychotic component of cannabis and has a variety of pharmacological effects, including anti-anxiety, antipsychotic, anti-spitting and anti-inflammatory properties.
Cannabidiol is non-toxic in non-converted cells, does not induce changes in food intake, does not induce systemic stiffness, does not affect physiological parameters (heart rate, blood pressure and body temperature), does not affect the transport of the gastrointestinal tract and does not change mental activity or mental function.
the FDA's urgency to develop effective therapies in this important patient group," said Armando Anido, Chairman and CEO of Zynerba.
to receive this title is an important milestone for us."
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