SMA Research Platform

Evidence graph for Spinal Muscular Atrophy

Biology-first target discovery
Christian Fischer / Bryzant Labs
14,766Targets
453Trials
84Drugs
7Datasets
6,987Sources
64,683Claims
72,052Evidence
29,649Hypotheses
DRUGapprovedsmall molecule

Omaveloxolone

Brand names: Skyclarys

Mechanism

NRF2 activator (KEAP1 inhibitor)

Related claims (5)

TypePredicateConfSource
gene expressionOmaveloxolone increases PGC1 levels selectively in SMA type I fibroblasts (demonstrated in vitro).59%10.64898/2026.03.17.712434
drug efficacyOmaveloxolone upregulates NRF2 target proteins in SMA type I fibroblasts (demonstrated in vitro).51%10.64898/2026.03.17.712434
drug efficacyOmaveloxolone produces a modest increase in SMN protein abundance selectively in SMA type I fibroblasts (demonstrated in vitro).51%10.64898/2026.03.17.712434
drug efficacySulforaphane exhibits superior neuroprotection compared to omaveloxolone with a favorable safety profile in methylmercury-induced ALS-like pathology.45%41649614
drug efficacyOmaveloxolone increases cell viability in SMA type I fibroblasts (demonstrated in vitro).45%10.64898/2026.03.17.712434

Off-Target Findings (0)

No Boltz-2 / Chai-1 off-target panel claims recorded for this drug.

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