ACTA2 Alliance
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acta2alliance.bsky.social
ACTA2 Alliance
@acta2alliance.bsky.social
Supporting families living with Multisystemic Smooth Muscle Dysfunction Syndrome #MSMDS #UltraRare #ACTA2 mutation https://www.zeffy.com/en-US/donation-form/6944a133-de97-4680-8675-ad6847c2d369
To the researchers & clinicians working tirelessly on this: THANK YOU 💜🩵💜

And to our community: you also have the chance to be part of this journey. By supporting research, you help discoveries like this one move closer to real treatments. This is how: bit.ly/MSMDS-Resear...
Shape the future of MSMDS Research
Welcome to the ACTA2 Alliance Foundation. We invite you to be part of a historic medical breakthrough for Multisystemic Smooth Muscle Dysfunction Syndrome (MSMDS), an ultra-rare genetic disease affect...
bit.ly
September 12, 2025 at 1:14 PM
We feel proud, grateful, and hopeful 💜
✨ Proud of how far this journey has come
✨ Grateful to researchers & clinicians leading the way
✨ Hopeful because we are getting closer to therapies our children need
September 12, 2025 at 1:13 PM
This is more than a step forward for MSMDS.
It’s a proof of concept that our rare disease can lead the way to treatments for other severe vascular diseases, like Moyamoya.
#vasculardiseases
September 12, 2025 at 1:13 PM
Using a custom-built CRISPR–Cas9 base editor, researchers corrected the ACTA2 mutation in a mouse model, prolonging survival and rescuing disease features in the brain, aorta & blood vessels.
September 12, 2025 at 1:12 PM
September 11, 2025 at 8:50 AM