Discover and read the best of Twitter Threads about #replagal
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SM presenting yesterday at HC Wainwright made a couple references to #Fabry study which prompted this thead
- No steroids have been used.
- Best data he's had the privilege to report
investor.sangamo.com/events/event-d…
$SGMO
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- No steroids have been used.
- Best data he's had the privilege to report
investor.sangamo.com/events/event-d…
$SGMO
1/
Fabry disease = abnormal a-Gal enzyme activity leading to Gb-3 and lyso-GB3 accumulation. Prevalence data varies widely given multiple attempts to quantify the impact of under-diagnosis. Kidney and cardiac disease are LT impacts
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ERT is the SOC for patients with #Fabry. The LT follow up studies for patients on ERT show that it does not stop disease progression and imposes a high treatment burden.
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