Hay Jon! A thread about A1AT
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Our most promising liver treatment at the moment is an RNA silencing drug that stops the SERPINA1 gene from being expressed at all, so no AAT protein made at all.
No Z protein made, the globules of previously-produced polymerized AAT in the hepatocytes clear in just a few months, and it seems that any fibrosis present starts to heal too, but we need more data on that. We’re recruiting for a registrational trial on that as we speak.
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It is interesting in general that skilled pulmonologists often don’t understand the liver aspect of the disease. My own pulmonologist at Duke once asked me if the infused augmentation therapy works on the liver disease.
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It is interesting in general that skilled pulmonologists often don’t understand the liver aspect of the disease. My own pulmonologist at Duke once asked me if the infused augmentation therapy works on the liver disease.
@jon-nyc said in Hay Jon! A thread about A1AT:
It is interesting in general that skilled pulmonologists often don’t understand the liver aspect of the disease. My own pulmonologist at Duke once asked me if the infused augmentation therapy works on the liver disease.
The ultra-specialization of medicine continues. This thread is full of physiology and biochemistry that I don't understand. That's not because I'm (too) stupid, it's because of things that I have not been exposed to in 45 years since I finished medical school. I have little doubt that if I started discussing pharmacology, analysis of arterial waveforms, these guys would be out of their field as well.
Nevertheless, I find myself frustrated by my ignorance.
Not to derail - well, maybe to derail a bit - there's a whole generation of gas passers who are trained in using ultrasonography for nerve blocks. These guys are sticking needles into places I would never have imagined, as recently as 10 years ago.
I'm such a dinosaur.
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@jon-nyc said in Hay Jon! A thread about A1AT:
It is interesting in general that skilled pulmonologists often don’t understand the liver aspect of the disease. My own pulmonologist at Duke once asked me if the infused augmentation therapy works on the liver disease.
The ultra-specialization of medicine continues. This thread is full of physiology and biochemistry that I don't understand. That's not because I'm (too) stupid, it's because of things that I have not been exposed to in 45 years since I finished medical school. I have little doubt that if I started discussing pharmacology, analysis of arterial waveforms, these guys would be out of their field as well.
Nevertheless, I find myself frustrated by my ignorance.
Not to derail - well, maybe to derail a bit - there's a whole generation of gas passers who are trained in using ultrasonography for nerve blocks. These guys are sticking needles into places I would never have imagined, as recently as 10 years ago.
I'm such a dinosaur.
