Is this a first? $LITE Stock trades down hard after a buyout is announced with a sizable premium?
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Fantastic News! Excited for the first approved treatment for HD.
We are thrilled to announce that the FDA has accepted for review the BLA for ifezuntirgene inilparvovec (AMT-130), an investigational #genetherapy for #HuntingtonsDisease. The application has been granted Priority Review with a PDUFA date of May 1, 2027. uniqure.gcs-web.com/news-rel…
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Would be great to see Mac versus T!
I captured this video in my lab to show the power of medicines called T-cell engagers.
Left: T cells move in and out of a cancer tumoroid, largely ignoring it.
Right: Add a T-cell engager, and the T cells attack. Once they recognize their targets, they tear through the tumor.
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Celebrating with my beautiful girls feeling blessed. Broncos & Roadster Event: icing on the cake!!!
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$QURE The updated 36-month dataset, now with 15 high-dose patients, actually shows 80% cUHDRS slowing, p=0.005 and 67% TFC slowing, p=0.011
More patients, better data.
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To all students (@OleMiss, @UCLA, @UCSB, etc). Please please be careful when you go out. Do not ingest “herbal” capsules, small tablets, etc. Kratom is extremely dangerous as it converts to 7-OH in your body. Some of these capsules contain pure 7-OH. It’s more than 10-times potent than morphine but depending on your body’s metabolism, it could generate an even stronger effect.
All students should carry Narcan with them, it will save lives, including your own. It’s on Amazon.
Please repost to your friends and to other universities.
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I think the board sometimes over thinks the strategy. They tried to get money so that the overhang is removed when data comes.
When in reality, everyone is waiting for data to validate the program and re-rate the stock. Which would support a raise.
I’d be more than happy to come in and help….
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Congratulations @ScholarRock! Great news for patients, more muscle 💪 is greatly needed!
BREAKING: Scholar Rock receives FDA approval for the first and only muscle-targeted treatment for all adults and children aged 2 and older living with spinal muscular atrophy (SMA) who are currently receiving a SMN2-targeted treatment. bit.ly/4gNc45r
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