Tuesday 13 December 2022

Hemophilia Occurs In About 1 Of Every 5,000 Male Births,

the condition is usually an inherited bleeding disorder in which the blood does not clot properly,


photograph Roger Fenton/Getty Images; Getty Images, perhaps the most famous suffer of the complaint was Alexei Nikolaevich, who inherited hemophilia from Queen Victoria, who was a carrier for hemophilia B, a rarer disease than hemophilia A, Queen Victoria is a good reminder that being a “blue blood” does not protect someone from having a bleeding disorder. Two of her daughters and her youngest son, Prince Leopold, inherited hemophilia from Queen Victoria, Leopold died after injuring his knee and hitting his head on a fall in 1884 when he was just 31 years old, four months before his son was born, Queen Victoria’s daughters passed on hemophilia to some of their children, and the condition earned the nickname “the royal disease” because so many of Victoria’s 26 adult grandchildren were carriers who married into royal families in Spain, Germany, and Russia, there is now some good and bad news for sufferers of this complaint, the good news,

photograph Firstpostlast Tuesday, the US Food and Drug Administration approved Hemgenix, a cutting-edge gene therapy designed to treat adults with hemophilia B, until now, typical treatments required routine injections to maintain sufficient levels of the missing protein in patients, but thanks to Hemgenix, sufferers of hemophilia B require a single IV infusion to be cured forever, “Today’s approval provides a new treatment option for patients with Hemophilia B and represents important progress in the development of innovative therapies for those experiencing a high burden of disease associated with this form of hemophilia,” Dr. Peter Marks, director of the FDA’s Centre for Biologics Evaluation and Research, said, and now the bad news, a price of $3.5 million, is being touted, making Hemgenix the most expensive drug ever, but according to a recent cost-effectiveness analysis by the Institute for Clinical and Economic Review, an independent nonprofit research organization, states a fair price for Hemgenix would be between $2.93 million and $2.96 million, which is still ridiculously prohibitive for the average person, hopefully a cheaper, much cheaper alternative will be found to help those with this life threating disease.


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