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Scientific Study conclusion
Scientific Study conclusion
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3D Mutation simulation
PACS1 FBR R203W HDAC6
PACS1 syndrome and the recurrent R203W mutation
Why ask whether existing drugs bind PACS1-R203W
Computational pipeline
Three robust hits at pocket #1
Pocket #19 — a cautionary tale about apparent super-targets
Pharmacophore — why these 3 drugs and not others
Independent validation campaigns
Reliability — honest assessment of every hypothesis
Clinical actionability — for cohorts and clinicians
Cost and open-science framing
Next steps and collaboration
Data and file access
References
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