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 Firstpost, last 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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