Hunter syndrome


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Hunter syndrome

[′hənt·ər ‚sin‚drōm]
(medicine)
An X-linked recessive disease in which a deficiency of the enzyme iduronate sulfatase leads to the accumulation of mucopolysaccharides in various body tissues, resulting in developmental abnormalities, skeletal deformations, mental retardation, and, in severe cases, early death. Also known as mucopolysaccharidosis II.
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NASDAQ: SGMO) has presented initial safety data from the CHAMPIONS Study, the Phase 1/2 clinical trial evaluating SB-913 for the treatment of mucopolysaccharidosis type II, a rare disease also known as Hunter syndrome, at the 2018 WORLDSymposium medical congress, the company said.
In November, 44-year-old Brian Madeux became the first person to have gene editing inside the body for a metabolic disease called Hunter syndrome that's caused by a bad gene.
In mid-November, Brian Madeux, a 44-year-old Phoenix man with a metabolic disease called Hunter syndrome, had just become the first person to try an experimental gene editing treatment.
8232;Dr David R Liu, from Harvard University, said: "The Hunter syndrome trial, led by a pioneering company, is another reminder that we have entered an era of genome editing," he said.
On Monday, in a medical first, Brian Madeux received an experimental in-body gene-editing treatment intended to cure him of Mucopolysaccharidoses II, known as MPS II or Hunter syndrome, a rare disorder that causes progressive damage to the body's cells.
com/4ae98919b52e43d8a8960e0e260feb0a/AP-Exclusive:-US-scientists-try-1st-gene-editing-in-the-body) AP reported today that researchers at UCSF Benioff Children's Hospital Oakland dripped new genes into the patient that will replace mutated ones causing his illness, known as Hunter syndrome.
On Monday, 44-year-old Brian Madeux AaAaAeAc who has a metabolic disease call Hunter syndrome AaAaAeAc received billions of copies of a corrective ge through an IV, along with a tool to cut his DNA in the relevant location.
Karen Johnson Pride of Britain Special Recognition 2016 After losing her two 12-year-old sons to the little-known Hunter Syndrome, Karen helped to raise PS2.
As long as Karen Johnson Pride of Britain Special Recognition 2016 After losing her two 12-year-old sons to the little-known Hunter Syndrome, Karen helped to raise PS2.
Also, mother Karen Johnson who lost two sons to Hunter Syndrome and almost lost her daughter to leukaemia.
Jasper Lee suffers from Hunter Syndrome, a rare disease in which he is missing an enzyme.
Treatment for rare diseases such as Pompe, Hunter Syndrome and nocturnal hemoglobinuria cost anything between US$300,000 to US$440,000 per patient.

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