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

arimoclomol

Brand names: Miplyffa

Mechanism

Hydroxylamine HSP70/HSP90 co-inducer. Preserves NMJ structure in SOD1-G93A ALS mice (PMID 22591194). Approved for Niemann-Pick type C (2024). Candidate SMA adjunct.

Approved indications

niemann-pick-c

Related claims (0)

No claims matched “arimoclomol” in free-text search.

Off-Target Findings (0)

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

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