SMA Research Platform

The SMA Research Platform tracks drugs across four relevance tiers — from officially approved SMA therapies to drugs approved for other conditions where computational or clinical evidence suggests SMA relevance. Every non-SMA-approved drug has a documented reason for inclusion.

Three FDA/EMA-approved treatments target the SMN pathway directly: nusinersen (antisense oligonucleotide, intrathecal), risdiplam (small-molecule splicing modifier, oral), and onasemnogene abeparvovec (AAV9 gene therapy, one-time IV). Pipeline drugs explore complementary approaches including muscle-enhancing (apitegromab, anti-myostatin), neuroprotective (riluzole, glutamate modulation), HDAC-mediated SMN2 upregulation (vorinostat), NMJ strengthening (pyridostigmine, amifampridine, argx-119, nmd-670), and oxidative-stress correction (dimethyl fumarate, omaveloxolone). Each drug entry links to DiffDock virtual screening results where available.