Drug Repurposing
COMPUTATIONALDrug repurposing means finding new therapeutic uses for existing approved drugs — bypassing the 10–15 years and $1–2B typically required for de novo drug development. Repurposed drugs have already passed safety trials, so clinical translation is dramatically faster: Phase I is often skipped and Phase II can start in 2–3 years rather than 10+.
The platform identifies SMA repurposing candidates through three convergent strategies: (1) Cross-disease mining — drugs approved or in trials for related neuromuscular diseases (ALS, Duchenne Muscular Dystrophy, SBMA, CMT) that share molecular targets with SMA; (2) ChEMBL bioactivity — known compounds with high pChEMBL values (≥ 6.0, corresponding to IC₅₀ ≤ 1 µM) against top-scored SMA targets; (3) Pathway overlap — compounds whose known mechanism overlaps with the actin dynamics, NMJ signaling, or autophagy/survival pathways dysregulated in SMA.
77
Total Candidates
17
Approved Drugs
9
Score ≥0.7
39
Cross-Disease
▶How does Repurposing Scoring work?
Each candidate is scored 0–1 based on target relevance, potency, clinical phase, and pathway convergence. Three convergent strategies: (1) Cross-disease mining — drugs approved/in trials for related neuromuscular diseases sharing SMA targets; (2) ChEMBL bioactivity — compounds with pChEMBL ≥ 6.0 (IC₅₀ ≤ 1 µM) against SMA targets; (3) Pathway overlap — mechanism overlaps SMA-relevant pathways.
Precedent: Valproic acid (HDAC inhibitor, epilepsy origin) was one of the first SMA trial compounds. Olesoxime reached Phase II. Riluzole (ALS) showed modest motor neuron protection in SMA models. The platform extends this approach computationally. Click any row to see full rationale, mechanism, and target link.
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| # | Compound | SMA Target | Score | Source | Phase | Outcome | Rationale |
|---|---|---|---|---|---|---|---|
| 1 | onasemnogene | SMN1 | 0.776 | cross disease | approved | success | onasemnogene shows success on SMN1 (approved). Gene therapy approved for spinal muscular atrophy treatment. |
| 2 | onasemnogene abeparvovec | SMN1 | 0.776 | cross disease | approved | success | onasemnogene abeparvovec shows success on SMN1 (approved). Third approved DMT; 11.4% of 2140 patients initiated treatment with onasemnogene abeparvovec in real-world cohort. |
| 3 | nusinersen | SMN2 | 0.768 | cross disease | approved | success | nusinersen shows success on SMN2 (approved). Approved treatment for SMA that increases SMN protein production through SMN2 splicing modification. |
| 4 | risdiplam | SMN2 | 0.768 | cross disease | approved | success | risdiplam shows success on SMN2 (approved). Second approved DMT; 24.0% of 2140 patients initiated treatment with risdiplam in real-world cohort. |
| 5 | onasemnogene abeparvovec (Zolgensma) | SMN1 | 0.761 | cross disease | approved | success | onasemnogene abeparvovec (Zolgensma) shows success on SMN1 (approved). Approved AAV gene therapy product with adequate toxicology characterized in ≤3-month studies |
| 6 | GNR-100 | SMN2 | 0.759 | cross disease | approved | success | GNR-100 shows success on SMN2 (approved). Generic nusinersen (GNR-100) demonstrates bioequivalence to reference nusinersen with identical oligonucleotide structure and comparable SMN-gene spli |
| 7 | nusinersen and risdiplam (pooled analysis) | SMN2 | 0.753 | cross disease | approved | partial success | nusinersen and risdiplam (pooled analysis) shows partial_success on SMN2 (approved). While both nusinersen and risdiplam achieved motor function improvements and high survival rates, clinical effectiveness was limited by variable outco |
| 8 | onasemnogene abeparvovec (OAV101 IT) | SMN1 | 0.726 | cross disease | phase3 | partial success | onasemnogene abeparvovec (OAV101 IT) shows partial_success on SMN1 (phase3). OAV101 IT demonstrated acceptable safety profile in treatment-experienced SMA patients with manageable adverse events, though hepatotoxicity and throm |
| 9 | Zolgensma | SMN1 | 0.701 | cross disease | approved | success | Zolgensma shows success on SMN1 (approved). Zolgensma represents a successful gene therapy option for SMA, though long-term effects and accessibility remain unresolved. |
| 10 | SMN-inducing drugs (general class) | SMN2 | 0.693 | cross disease | approved | success | SMN-inducing drugs (general class) shows success on SMN2 (approved). SMN-inducing drugs improved motor function and fatigability in SMA patients with concurrent restoration of sensory-motor circuit function measured by |
| 11 | riluzole | multiple | 0.685 | cross disease | approved | success | riluzole shows success on GLUTAMATE SIGNALING (approved). Riluzole pharmacologically rescued motor neuron survival in SALS-derived neurons, demonstrating drug efficacy in patient model. |
| 12 | tofersen | multiple | 0.685 | cross disease | approved | success | tofersen shows success on SOD1 MRNA (approved). Tofersen is the first approved therapy for SOD1-ALS with biomarker evidence of target engagement showing reduced neurofilament levels and other neural |
| 13 | eteplirsen (Luxturna) | RPE65 | 0.681 | cross disease | approved | success | eteplirsen (Luxturna) shows success on RPE65 (approved). Approved AAV gene therapy product with adequate toxicology characterized in ≤3-month studies |
| 14 | edaravone | multiple | 0.676 | cross disease | approved | partial success | edaravone shows partial_success on OXIDATIVE STRESS (approved). Edaravone demonstrates modest neuroprotective effects in preclinical models and a subset of ALS patients, but with inconsistent results and limited cl |
| 15 | eteplirsen | multiple | 0.670 | cross disease | approved | partial success | eteplirsen shows partial_success on DMD EXON 51 (approved). Eteplirsen received accelerated approval for DMD but lacks convincing functional evidence of slowing disease progression and has limited geographic ap |
| 16 | SMN-inducing drugs | SMN2 | 0.663 | cross disease | approved | success | SMN-inducing drugs shows success on SMN2 (approved). SMN-inducing drugs improved motor function and fatigability in ambulatory type 3 SMA patients and mouse models, with improvements correlated to increa |
| 17 | SMN-inducing drugs (unspecified) | SMN2 | 0.663 | cross disease | approved | partial success | SMN-inducing drugs (unspecified) shows partial_success on SMN2 (approved). SMN-inducing drugs improve motor function and fatigability in type 3 SMA patients and models, with efficacy monitored by Hoffmann reflex assay measuri |
| 18 | CHEMBL3752910 | STMN2 | 0.586 | chembl bioactivity | preclinical | — | CHEMBL3752910 has pChEMBL=8.4 on STMN2 (target score: 0.336). SMILES: Cc1ccc(C(=O)Nc2cccc(C(F)(F)F)c2)cc1Nc1nc(-c2cccnc2)nc2c1cnn2... |
| 19 | CHEMBL5759269 | UBA1 | 0.582 | chembl bioactivity | preclinical | — | CHEMBL5759269 has pChEMBL=7.3 on UBA1 (target score: 0.437). SMILES: NS(=O)(=O)OC[C@H]1C[C@@H](Nc2ccnc3cc(-c4ccccc4)nn23)[C@H](O)... |
| 20 | CHEMBL5177755 | UBA1 | 0.578 | chembl bioactivity | preclinical | — | CHEMBL5177755 has pChEMBL=7.2 on UBA1 (target score: 0.437). SMILES: COC(=O)N1CCC2(CC1)C[C@H](Nc1ncnc3c1nnn3[C@@H]1C[C@@H](COS(N)... |
| 21 | Luxturna | RPE65 | 0.576 | cross disease | approved | success | Luxturna shows success on RPE65 (approved). Luxturna is an approved gene therapy for rare conditions, though prohibitive pricing limits accessibility. |
| 22 | CHEMBL5653589 | UBA1 | 0.570 | chembl bioactivity | preclinical | — | CHEMBL5653589 has pChEMBL=7.0 on UBA1 (target score: 0.437). SMILES: Cc1ccc(C(=O)Nc2cccc(C(F)(F)F)c2)cc1Nc1nc(-c2cccnc2)nc2nn(C)c... |
| 23 | CHEMBL5175806 | UBA1 | 0.536 | chembl bioactivity | preclinical | — | CHEMBL5175806 has pChEMBL=6.3 on UBA1 (target score: 0.437). SMILES: CCOc1cccc(F)c1C#Cc1cn([C@@H]2O[C@H](CNS(N)(=O)=O)[C@@H](O)[C... |
| 24 | CHEMBL5793677 | UBA1 | 0.532 | chembl bioactivity | preclinical | — | CHEMBL5793677 has pChEMBL=6.3 on UBA1 (target score: 0.437). SMILES: Cc1cc(N[C@@H]2C[C@H](COS(N)(=O)=O)[C@@H](O)[C@H]2O)n2nc(-c3c... |
| 25 | CHEMBL5743820 | UBA1 | 0.532 | chembl bioactivity | preclinical | — | CHEMBL5743820 has pChEMBL=6.3 on UBA1 (target score: 0.437). SMILES: NS(=O)(=O)OC[C@H]1C[C@@H](Nc2cc(Cl)nc3cc(-c4ccccc4)nn23)[C@H... |
| 26 | CHEMBL5866556 | UBA1 | 0.532 | chembl bioactivity | preclinical | — | CHEMBL5866556 has pChEMBL=6.3 on UBA1 (target score: 0.437). SMILES: NS(=O)(=O)OC[C@H]1C[C@@H](Nc2cc(Cl)nc3cc(-c4ccc(Br)cc4)nn23)... |
| 27 | CHEMBL5799754 | UBA1 | 0.532 | chembl bioactivity | preclinical | — | CHEMBL5799754 has pChEMBL=6.3 on UBA1 (target score: 0.437). SMILES: NS(=O)(=O)OC[C@H]1C[C@@H](Nc2cc(Cl)nc3cc(-c4cccc5ccccc45)nn2... |
| 28 | CHEMBL5440318 | UBA1 | 0.529 | chembl bioactivity | preclinical | — | CHEMBL5440318 has pChEMBL=6.2 on UBA1 (target score: 0.437). SMILES: C=S(C)(=O)N1CC(=O)N[C@@H](CN(CC(=O)N[C@H](CCCCN)CN(CC(=O)N[C... |
| 29 | CHEMBL5433769 | UBA1 | 0.526 | chembl bioactivity | preclinical | — | CHEMBL5433769 has pChEMBL=6.1 on UBA1 (target score: 0.437). SMILES: C=S(C)(=O)N(CC(=O)N[C@H](CC(C)C)CN(CC(=O)N[C@H](CCCCN)CN(CC(... |
| 30 | CHEMBL1359097 | SMN2 | 0.522 | chembl bioactivity | preclinical | — | CHEMBL1359097 has pChEMBL=8.3 on SMN2 (target score: 0.208). SMILES: O=C(c1ccc(SCc2cccc(Br)c2)c([N+](=O)[O-])c1)N1CCN(c2ccccn2)CC... |
| 31 | CHEMBL1409365 | SMN2 | 0.519 | chembl bioactivity | preclinical | — | CHEMBL1409365 has pChEMBL=8.3 on SMN2 (target score: 0.208). SMILES: CC(C)(C)C1CCC(OC(=O)C[N+]2(C)CCCCC2)CC1.[I-] |
| 32 | CHEMBL1339757 | SMN2 | 0.519 | chembl bioactivity | preclinical | — | CHEMBL1339757 has pChEMBL=8.3 on SMN2 (target score: 0.208). SMILES: Cc1ccc(NC(=O)Nc2c(C)n(C)n(-c3ccccc3)c2=O)cc1C |
| 33 | nusinersen-like antisense oligonucleotide (intracerebroventricular administration) | SMN2 | 0.512 | cross disease | preclinical | success | nusinersen-like antisense oligonucleotide (intracerebroventricular administration) shows success on SMN2 (preclinical). ICV administration of nusinersen-like ASO restored SMN2 splicing, improved survival and motor function, and prevented lymphoid organ pathology includi |
| 34 | nusinersen-like antisense oligonucleotide (subcutaneous administration) | SMN2 | 0.509 | cross disease | preclinical | success | nusinersen-like antisense oligonucleotide (subcutaneous administration) shows success on SMN2 (preclinical). Subcutaneous administration of nusinersen-like ASO restored SMN2 splicing, improved survival and motor function, and prevented lymphoid pathology in s |
| 35 | DG9-PMO | SMN2 | 0.509 | cross disease | preclinical | partial success | DG9-PMO shows partial_success on SMN2 (preclinical). DG9-conjugated PMO demonstrates improved survival, motor coordination, muscle pathology mitigation, and CNS distribution via subcutaneous injection co |
| 36 | F18MOE | SMN2 | 0.503 | cross disease | preclinical | partial success | F18MOE shows partial_success on SMN2 (preclinical). F18MOE achieves on-target SMN2 splicing modulation but causes widespread off-target exon skipping and transcriptome dysregulation via sequence-depende |
| 37 | CHEMBL1575581 | SMN2 | 0.499 | chembl bioactivity | preclinical | — | CHEMBL1575581 has pChEMBL=7.9 on SMN2 (target score: 0.208). SMILES: Cc1ccc2c(N)c(C#N)sc2n1 |
| 38 | CRISPR-Q (CasRx-based transcript knockdown) | SMN1 | 0.496 | cross disease | preclinical | success | CRISPR-Q (CasRx-based transcript knockdown) shows success on SMN1 (preclinical). CRISPR-Q system achieved simultaneous knockdown of tardbp and tardbpl to model amyotrophic lateral sclerosis in zebrafish. |
| 39 | R6G-PMO | SMN2 | 0.488 | cross disease | preclinical | partial success | R6G-PMO shows partial_success on SMN2 (preclinical). Benchmark cell-penetrating peptide-conjugated PMO shows lesser efficacy than DG9-PMO in rescue of phenotype in SMA mice. |
| 40 | CHEMBL555257 | DNMT3B | 0.483 | chembl bioactivity | preclinical | — | CHEMBL555257 has pChEMBL=6.6 on DNMT3B (target score: 0.309). SMILES: O=C(O)[C@@H]1C[C@H](SC[C@H]2O[C@@H](n3cnc4c(NCCc5ccc(-c6cccc... |
| 41 | CHEMBL418052 | DNMT3B | 0.480 | chembl bioactivity | preclinical | — | CHEMBL418052 has pChEMBL=6.5 on DNMT3B (target score: 0.309). SMILES: Nc1ncnc2c1ncn2[C@@H]1O[C@H](CSCC[C@H](N)C(=O)O)[C@@H](O)[C@H... |
| 42 | CHEMBL552309 | DNMT3B | 0.480 | chembl bioactivity | preclinical | — | CHEMBL552309 has pChEMBL=6.5 on DNMT3B (target score: 0.309). SMILES: Nc1ncnc2c1ccn2[C@@H]1O[C@H](CSCC[C@H](N)C(=O)O)[C@@H](O)[C@H... |
| 43 | CHEMBL557902 | DNMT3B | 0.480 | chembl bioactivity | preclinical | — | CHEMBL557902 has pChEMBL=6.5 on DNMT3B (target score: 0.309). SMILES: Nc1ncnc2c1ncn2[C@@H]1O[C@H](CS[C@@H]2CN[C@H](C(=O)O)C2)[C@@H... |
| 44 | CHEMBL1339741 | SMN2 | 0.469 | chembl bioactivity | preclinical | — | CHEMBL1339741 has pChEMBL=7.3 on SMN2 (target score: 0.208). SMILES: CC(=O)Nc1ccc(Nc2nc(C)c([N+](=O)[O-])c(Nc3ccc(NC(C)=O)cc3)n2)... |
| 45 | CHEMBL2106789 | DNMT3B | 0.469 | chembl bioactivity | preclinical | — | CHEMBL2106789 has pChEMBL=6.3 on DNMT3B (target score: 0.309). SMILES: C[C@@H]1O[C@@H](CC(=O)O)CC2=C1C(=O)c1c(O)cccc1C2=O |
| 46 | CHEMBL1497549 | SMN2 | 0.467 | chembl bioactivity | preclinical | — | CHEMBL1497549 has pChEMBL=7.2 on SMN2 (target score: 0.208). SMILES: O=C(NC(Nc1nc2ccc([N+](=O)[O-])cc2s1)(C(F)(F)F)C(F)(F)F)c1ccc... |
| 47 | AAV9-IGHMBP2-P546 | IGHMBP2 | 0.465 | cross disease | preclinical | success | AAV9-IGHMBP2-P546 shows success on IGHMBP2 (preclinical). P546-promoter AAV9-IGHMBP2 construct demonstrated superior long-term efficacy and safety profile, effectively rescuing SMARD1 pathological hallmarks i |
| 48 | CHEMBL560105 | DNMT3B | 0.465 | chembl bioactivity | preclinical | — | CHEMBL560105 has pChEMBL=6.2 on DNMT3B (target score: 0.309). SMILES: NC(CCSC[C@H]1O[C@@H](n2cnc3c(NCCCc4ccccc4)ncnc32)[C@H](O)[C@... |
| 49 | CHEMBL555352 | DNMT3B | 0.465 | chembl bioactivity | preclinical | — | CHEMBL555352 has pChEMBL=6.2 on DNMT3B (target score: 0.309). SMILES: NC(CCSC[C@H]1O[C@@H](n2cnc3c(NCCc4ccc(-c5ccccc5)cc4)ncnc32)[... |
| 50 | CHEMBL1477410 | SMN2 | 0.459 | chembl bioactivity | preclinical | — | CHEMBL1477410 has pChEMBL=7.1 on SMN2 (target score: 0.208). SMILES: CN(C)c1ccc(C(c2nnnn2CCOC(=O)Nc2ccc(Cl)cc2)N2CCOCC2)cc1 |
| 51 | CHEMBL560505 | DNMT3B | 0.457 | chembl bioactivity | preclinical | — | CHEMBL560505 has pChEMBL=6.0 on DNMT3B (target score: 0.309). SMILES: COc1cc(CNc2ncnc3c2ncn3[C@@H]2O[C@H](CSCCC(N)C(=O)O)[C@@H](O)... |
| 52 | CHEMBL1564869 | DNMT3B | 0.457 | chembl bioactivity | preclinical | — | CHEMBL1564869 has pChEMBL=6.0 on DNMT3B (target score: 0.309). SMILES: O=C(O)[C@H](Cc1c[nH]c2ccccc12)N1C(=O)c2ccccc2C1=O |
| 53 | CHEMBL559283 | DNMT3B | 0.456 | chembl bioactivity | preclinical | — | CHEMBL559283 has pChEMBL=6.0 on DNMT3B (target score: 0.309). SMILES: O=C(O)[C@@H]1C[C@H](SC[C@H]2O[C@@H](n3cnc4c(NCCc5ccc(-c6cccc... |
| 54 | CHEMBL560045 | DNMT3B | 0.454 | chembl bioactivity | preclinical | — | CHEMBL560045 has pChEMBL=6.0 on DNMT3B (target score: 0.309). SMILES: Cc1nc(N)c2ncn([C@@H]3O[C@H](CSCCC(N)C(=O)O)[C@@H](O)[C@H]3O)... |
| 55 | CHEMBL556265 | DNMT3B | 0.454 | chembl bioactivity | preclinical | — | CHEMBL556265 has pChEMBL=6.0 on DNMT3B (target score: 0.309). SMILES: NC(CCSC[C@H]1O[C@@H](n2cnc3c(NCc4ccccc4)ncnc32)[C@H](O)[C@@H... |
| 56 | AAV9-IGHMBP2-CBA | IGHMBP2 | 0.450 | cross disease | preclinical | partial success | AAV9-IGHMBP2-CBA shows partial_success on IGHMBP2 (preclinical). CBA-promoter construct rescued SMARD1 phenotype and improved survival, body weight, and motor function, but showed inferior long-term efficacy versus |
| 57 | CHEMBL1458133 | SMN2 | 0.449 | chembl bioactivity | preclinical | — | CHEMBL1458133 has pChEMBL=6.9 on SMN2 (target score: 0.208). SMILES: O=C(COC(=O)CCCN1C(=O)c2cccc3cccc(c23)C1=O)Nc1cccc(Br)c1 |
| 58 | CHEMBL1575524 | SMN2 | 0.444 | chembl bioactivity | preclinical | — | CHEMBL1575524 has pChEMBL=6.8 on SMN2 (target score: 0.208). SMILES: COc1ccc(-n2c(=O)c3ccccc3n(CC(=O)Nc3cc(C)on3)c2=O)cc1OC |
| 59 | CUG codon mutation (base editing approach) | multiple | 0.435 | cross disease | preclinical | success | CUG codon mutation (base editing approach) shows success on C9ORF72 RAN TRANSLATION INITIATION CODON (preclinical). Blocking RAN translation-derived DPR production alleviates behavioral deficits, motor neuron loss, neuroinflammation, p-TDP-43 inclusions, and increas |
| 60 | MSC-derived small extracellular vesicles (MSC-sEVs) | multiple | 0.435 | cross disease | preclinical | partial success | MSC-derived small extracellular vesicles (MSC-sEVs) shows partial_success on MULTIPLE NEUROPROTECTIVE PATHWAYS (preclinical). Intranasal MSC-sEVs delayed functional deterioration and prolonged mild impairment stage but did not significantly extend overall survival in ALS mous |
| 61 | AAVrh74.tMCK.NT-3 | multiple | 0.435 | cross disease | preclinical | success | AAVrh74.tMCK.NT-3 shows success on NT-3 (NEUROTROPHIN 3) (preclinical). AAVrh74.tMCK.NT-3 gene therapy significantly improved grip strength, rotarod performance, electrophysiological parameters, and NMJ denervation while r |
| 62 | CHEMBL1379627 | SMN2 | 0.434 | chembl bioactivity | preclinical | — | CHEMBL1379627 has pChEMBL=6.6 on SMN2 (target score: 0.208). SMILES: O=C(Nc1ccc(C(F)(F)F)cc1)c1cnc(N2CCN(c3ccccn3)CC2)c2ccccc12 |
| 63 | N-of-1 ASO for TTD-A (p52ΔC-inducing) | GTF2H4 | 0.432 | cross disease | preclinical | partial success | N-of-1 ASO for TTD-A (p52ΔC-inducing) shows partial_success on GTF2H4 (preclinical). ASO-induced p52 C-terminal truncation successfully stabilized TFIIH complex in p8-deficient patient cells, providing proof-of-concept for N-of-1 ASO t |
| 64 | CHEMBL4125910 | LY96 | 0.432 | chembl bioactivity | preclinical | — | CHEMBL4125910 has pChEMBL=6.2 on LY96 (target score: 0.244). SMILES: N[C@@H](Cc1ccccc1)C(=O)N[C@@H](CO)C(=O)N[C@@H](CO)C(=O)N[C@@... |
| 65 | clomipramine | SOD1 | 0.431 | cross disease | preclinical | partial success | clomipramine shows partial_success on SOD1 (preclinical). Clomipramine ameliorates lifespan reduction, improves climbing abilities, and mitigates genomic instability and inflammation in Drosophila SOD1-ALS mo |
| 66 | melatonin + edaravone combination | multiple | 0.414 | cross disease | preclinical | success | melatonin + edaravone combination shows success on IGF-1 SIGNALING, OXIDATIVE STRESS, NEUROINFLAMMATION (preclinical). Melatonin and edaravone combination exhibited synergistic neuroprotective effects surpassing individual treatments in reducing oxidative stress, infla |
| 67 | CHEMBL1604526 | SMN2 | 0.414 | chembl bioactivity | preclinical | — | CHEMBL1604526 has pChEMBL=6.2 on SMN2 (target score: 0.208). SMILES: Cn1nc(C(=O)NCCN2CCOCC2)c2ccccc2c1=O |
| 68 | CHEMBL1557815 | SMN2 | 0.412 | chembl bioactivity | preclinical | — | CHEMBL1557815 has pChEMBL=6.2 on SMN2 (target score: 0.208). SMILES: Cc1cccc(NC(=O)Cn2cc(C(=O)c3ccco3)c3ccccc32)c1 |
| 69 | CHEMBL1319158 | SMN2 | 0.409 | chembl bioactivity | preclinical | — | CHEMBL1319158 has pChEMBL=6.1 on SMN2 (target score: 0.208). SMILES: Cc1cc(C)c(C#N)c(SCc2nc(O)c3ccccc3n2)n1 |
| 70 | RC-1001 | multiple | 0.405 | cross disease | preclinical | partial success | RC-1001 shows partial_success on DYSTROPHIN (preclinical). RC-1001 PPMO treatment showed dose-independent improvements in respiratory function with dystrophin restoration and reduced inflammation in respirator |
| 71 | melatonin (MLT) | multiple | 0.405 | cross disease | preclinical | partial success | melatonin (MLT) shows partial_success on IGF-1 SIGNALING PATHWAY (preclinical). Melatonin at 5 and 10 mg/kg doses dose-dependently mitigated MEME-induced neurotoxicity, improved motor function, and activated IGF-1 signaling in a r |
| 72 | MK-8722 | multiple | 0.405 | cross disease | preclinical | partial success | MK-8722 shows partial_success on AMPK (preclinical). Direct AMPK activation via MK-8722 safely improves muscle function, mitochondrial health, and reduces dystrophic pathology in a mutation-independent m |
| 73 | baricitinib | multiple | 0.405 | cross disease | preclinical | partial success | baricitinib shows partial_success on JAK/STAT PATHWAY (preclinical). Baricitinib identified as component of promising combinatorial therapy with memantine and riluzole for SALS in iPSC model. |
| 74 | memantine | multiple | 0.405 | cross disease | preclinical | partial success | memantine shows partial_success on NMDA RECEPTOR (preclinical). Memantine identified as component of promising combinatorial therapy with baricitinib and riluzole for SALS in iPSC model. |
| 75 | IMD-0354 | multiple | 0.405 | cross disease | preclinical | partial success | IMD-0354 shows partial_success on NF-ΚB/IRF5 PATHWAY (preclinical). Absence of IRF5 expression in macrophages significantly amplifies the neuroprotective efficacy of IMD-0354 against TDP-25-induced neurodegeneration, s |
| 76 | CHEMBL1516729 | SMN2 | 0.404 | chembl bioactivity | preclinical | — | CHEMBL1516729 has pChEMBL=6.0 on SMN2 (target score: 0.208). SMILES: C=CCn1c(C)cc(C(=O)CSc2nc3ccccc3[nH]2)c1C |
| 77 | edaravone (EDR) | multiple | 0.390 | cross disease | preclinical | partial success | edaravone (EDR) shows partial_success on OXIDATIVE STRESS/ANTIOXIDANT (preclinical). Edaravone alone showed neuroprotective effects; combination with melatonin exhibited synergistic effects exceeding individual treatment efficacy. |