Discover and read the best of Twitter Threads about #Fabry

Most recents (2)

$SGMO status going into C22
Two synthetic gene transfer programs:
- HemA now run by $PFE P3. Hold for protocol amendment. Expect timing update for full analysis in next couple months.
- #Fabry owned program good data potential first/best in class
No further programs.
1/
$SGMO development focus leveraging ZF technology shows evolution from old four platforms to new integrated tech message. ZF tech facilitates both ex vivo and in vivo genomic medicines. Examples are CAR Tregs cell therapy and ZFP-TF CNS regulation.
2/ ImageImageImageImage
Cell therapy pipeline:
-Sickle cell collab with $SNY lead indication (see next)
-$GILD collab programs on hold. No update for a while but likely tied to durability issues seen at $ALLO $CRSP $DTIL
-Steadfast study is #POC for CAR Tregs with significant pipeline to follow
3/ ImageImageImageImage
Read 9 tweets
#WeekendLecture
#GeneticsAndStroke

#Genetics is involved in several #stroke related conditions, such as small vessel disease #SVD, aneurysms, dissections, cavernomas, and intracranial #hemorrhages

Check this 🧵!
Prepared by @J_Rosales_MD
Edited by @interneurona Image
#Weekendlecture

#Genetic & #SVD
🧬🧠

✅1,5% of cerebral #SVD are #monogenic disorders
✅AR, AD or X-linked
☝🏼Absence of family history doesn't rule out diagnosis because of
👉de novo mutation
👉reduced expression or
👉incomplete penetrance
ahajournals.org/doi/10.1161/ST…
#Weekendlecture

#Genetic & #SVD

When to #suspect?🕵️‍♀️

✅early onset stroke without vascular RFs
✅systemic features and neuroimaging of an specific monogenic disorder
✅consanguinity
✅negative work up for other causes of SVD

👉ahajournals.org/doi/10.1161/ST… Image
Read 15 tweets

Related hashtags

Did Thread Reader help you today?

Support us! We are indie developers!


This site is made by just two indie developers on a laptop doing marketing, support and development! Read more about the story.

Become a Premium Member ($3.00/month or $30.00/year) and get exclusive features!

Become Premium

Too expensive? Make a small donation by buying us coffee ($5) or help with server cost ($10)

Donate via Paypal Become our Patreon

Thank you for your support!