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3D visualization of the pocket mutation

3D visualization of the pocket mutation

3D PACS1 with drug

PACS1-FBR ↔ HDAC6-CD2

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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

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