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![aditharun_ Avatar](https://lunarcrush.com/gi/w:24/cr:twitter::1037546970154459136.png) Adith Arun [@aditharun_](/creator/twitter/aditharun_) on x XXX followers
Created: 2025-07-23 00:46:00 UTC

$srpt's viral vector appears to trigger liver failure in a small subset of patients that led to X deaths. And the 3rd death in the limb girdle is probably related.  What immunosuppression regimen can $srpt use to prevent immune cascade? Do we have to immunosupress everyone? What supporting therapies can we use to prevent acute liver failure if even mild liver enzyme spikes are observed? How can we identify which minority of patients will experience serious liver injury?

The viral vector is the common thread here. It is reasonable that FDA revoked platform designation for $srpt viral vector in light of this. The X DMD patients were hospitalized < X months from infusion and died of liver failure and the third (limb-girdle patient) died within X months with elevated liver enzymes < X months after infusion. This time course is in line with the X month window after infusion where the viral vector can cause liver damage [*]

All EHR data from the ~900 patients dosed with elevidys needs to be closely analyzed (liver labs, CBC, echo, clinical status, etc.). What other toxicities are lurking that we should be aware of? What are the time course of symptoms and is it all coming from the viral vector? 

@adamfeuerstein and @statnews have covered the communication failure from both $srpt and FDA. This must be fixed for all future drug development. Why is $srpt management not being more proactive?


XXX engagements

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**Related Topics**
[cascade](/topic/cascade)
[$srpt](/topic/$srpt)
[$srpts](/topic/$srpts)
[arun](/topic/arun)
[sarepta therapeutics inc](/topic/sarepta-therapeutics-inc)
[stocks healthcare](/topic/stocks-healthcare)

[Post Link](https://x.com/aditharun_/status/1947820457685663808)

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aditharun_ Avatar Adith Arun @aditharun_ on x XXX followers Created: 2025-07-23 00:46:00 UTC

$srpt's viral vector appears to trigger liver failure in a small subset of patients that led to X deaths. And the 3rd death in the limb girdle is probably related. What immunosuppression regimen can $srpt use to prevent immune cascade? Do we have to immunosupress everyone? What supporting therapies can we use to prevent acute liver failure if even mild liver enzyme spikes are observed? How can we identify which minority of patients will experience serious liver injury?

The viral vector is the common thread here. It is reasonable that FDA revoked platform designation for $srpt viral vector in light of this. The X DMD patients were hospitalized < X months from infusion and died of liver failure and the third (limb-girdle patient) died within X months with elevated liver enzymes < X months after infusion. This time course is in line with the X month window after infusion where the viral vector can cause liver damage [*]

All EHR data from the ~900 patients dosed with elevidys needs to be closely analyzed (liver labs, CBC, echo, clinical status, etc.). What other toxicities are lurking that we should be aware of? What are the time course of symptoms and is it all coming from the viral vector?

@adamfeuerstein and @statnews have covered the communication failure from both $srpt and FDA. This must be fixed for all future drug development. Why is $srpt management not being more proactive?

XXX engagements

Engagements Line Chart

Related Topics cascade $srpt $srpts arun sarepta therapeutics inc stocks healthcare

Post Link

post/tweet::1947820457685663808
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