Arcadia’s treatment for rare female pediatric genetic disorders
Acadia Pharmaceuticals has received FDA approval for Daybue (Trofinetide), a treatment for Rett Syndrome.
Arcadia announced on the 11th that Dayview has been approved for the treatment of Rett Syndrome in children and adults over the age of two. Additional priority screening vouchers have been secured for pediatric indications, and Arcadia plans to release the product in April.
Rett Syndrome is a disease in which the growth of brain function is impaired because the X chromosome is not activated due to a mutation in the gene called MeCP2 (Methyl-CpG binding protein 2). It affects 1 in 10,000 to 15,000 girls worldwide. It is estimated that there are 6,000 to 9,000 patients in the United States, and 4,500 patients have been diagnosed.
It is a genetic disease that develops normally from 6 to 18 months after birth, then develops language and movement disorders, and goes through a regression phase in which acquired functions are lost from 1 to 4 years after birth. X chromosome abnormalities occur mainly in the opposite sex. To date, there is no cure.
Arcadia has been co-developed by Australia’s Neuren Pharmaceuticals, which bought the US copyright in 2018.
Dayview is looking into the possibility of treatment for fragile (fragile) X chromosome syndrome other than Rett syndrome. Fragile (fragile) X chromosome syndrome is a genetic disorder of mental retardation that occurs in men, as opposed to Rett syndrome, which occurs in women.
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