CitizenVectron Posted July 21, 2022 Share Posted July 21, 2022 Transformational therapy cures haemophilia B WWW.BBC.COM A patient on the trial says his life now feels "completely normal", having grown up fearing getting hurt. This is the less-common and less-severe version of hemophilia, but it's great news. It appears the gene therapy is a one-time treatment that theoretically could last a lifetime, but probably at least a decade. 2 Quote Link to comment Share on other sites More sharing options...
Guest Posted July 21, 2022 Share Posted July 21, 2022 Quote Link to comment Share on other sites More sharing options...
CayceG Posted July 21, 2022 Share Posted July 21, 2022 104 years too late for Alexei Romanov. RIP. For real, a guy I knew when I was younger has a kid with hemophilia. I don't know if it's B or not, but I really hope he's able to get this therapy. Quote Link to comment Share on other sites More sharing options...
Keyser_Soze Posted July 21, 2022 Share Posted July 21, 2022 Do you think the guy on that Hemlibra commercial is actually a hemophiliac? Quote Link to comment Share on other sites More sharing options...
Jwheel86 Posted July 21, 2022 Share Posted July 21, 2022 The SMA community sends its regards. Biomechanically this is exactly the same as Zolgensma, except they can dose adults without the AAV virus killing their liver, probably because the liver is the intended target unlike SMA where it needs to cross the blood brain barrier to hit motor neurons, very cool. We still have no idea how long Zolgensma will last but I've saw a video the other day of a kid, who genetically should be on a vent or dead, running around with no SMA symptoms. The mRNA splicer in my fridge is cooler though. Quote How much will the gene therapies cost? Current clotting factor injections can cost between £150,000 and £200,000 per patient per year Zolgensma costs $2.1m so I suspect it'll be the same. 1 Quote Link to comment Share on other sites More sharing options...
Jwheel86 Posted August 12, 2022 Share Posted August 12, 2022 Novartis reports Zolgensma caused two deaths from liver failure WWW.REUTERS.COM Novartis AG on Thursday reported two patient fatalities due to acute liver failure following treatment with Zolgensma gene therapy used to treat spinal muscular atrophy. Exact same technology as the Hemophilia treatment. Quote Link to comment Share on other sites More sharing options...
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