An autonomous, multi-method computational engine that screens, ranks, and stress-tests therapeutic hypotheses for SMA — combining molecular docking, structure prediction, ADMET modeling, and automated evidence synthesis. Every result here is a computational prediction or hypothesis, not a wet-lab-validated finding. We surface contradicted and negative results alongside promising ones, because rigorous self-correction is the point.
A correction to our earlier 4-AP framing Two earlier internal framings of 4-aminopyridine were too broad. Additional DiffDock runs support a narrower, mechanism...
What the 2026 SMA Congress told us The 5th International Scientific Congress on SMA (Budapest, 11–14 March 2026) reframed the agrin-LRP4-MuSK-DOK7-RAPSN NMJ axi...
**Every result below is a computational hypothesis — a prediction from simulation and structure modeling, not a wet-lab-validated finding, and not a statement o...
Scholar Rock announced 8 May 2026 that the U.S. Food and Drug Administration has accepted its Biologics License Application (BLA) for apitegromab. The Prescript...
The thesis Fasudil alone is a single-mechanism cytoskeletal rescue. The 2026 SMA Congress (Budapest) and the Cure SMA 2025 meeting together handed the field two...
Menduti et al. (Experimental and Molecular Medicine, 2026) report that haloperidol — a D2 dopamine receptor antagonist FDA-approved since 1967 for psychosis — i...
Osseni et al. (Brain, 2026) report that combining SMN2 splice modifiers (such as antisense oligonucleotides inducing exon 7 inclusion) with systemic HDAC6 inhib...
What was reported At the 5th International Scientific Congress on SMA (Budapest, 11–14 March 2026), Crawford, Sansone, Hagenacker and colleagues from Biogen pre...
Cross-database analysis of FDA-approved drugs against the HDAC6 mechanism validated by Osseni et al. (Brain 2026) for SMA identifies Givinostat (Duvyzat, Italfa...