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
DRUGpreclinicalsmall molecule

gv-58

Brand names: GV-58

Mechanism

Selective Cav2 calcium-channel gating modifier. Stabilizes open state → enhances Ca2+ influx into motor nerve terminals → boosts ACh release. In severe SMA mice, GV-58 + DAP combination restored quantal content ~44% and improved muscle strength.

Approved indications

SMA preclinical (severe SMNΔ7 mouse)

Related claims (5)

TypePredicateConfSource
motor functionAcute subcutaneous administration of GV-58 significantly increases behavioral measures of muscle strength in SMNΔ7 mice.53%36757138
motor functionThe combination of GV-58 and 3,4-DAP significantly increases behavioral measures of muscle strength when administered subcutaneously in SMNΔ7 mice.53%36757138
drug targetGV-58 is a calcium channel gating modifier that acts as a drug target for SMA treatment.53%36757138
drug efficacyGV-58, a calcium channel gating modifier, restores neuromuscular transmission in SMNΔ7 mice when applied in vitro.45%36757138
drug efficacyThe combination of GV-58 and 3,4-DAP (a potassium channel antagonist) significantly restores neuromuscular transmission in SMNΔ7 mice.45%36757138

Off-Target Findings (0)

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

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