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
DRUGphase2small molecule

pyridostigmine

Brand names: Mestinon

Mechanism

Acetylcholinesterase inhibitor. Improves neuromuscular transmission at the NMJ.

Related claims (5)

TypePredicateConfSource
motor functionsEMG parameters indicate enhanced low-threshold (LT) motor unit (MU) function in upper-extremity muscles of patients with SMA treated with pyridostigmine.59%37595479
drug efficacyPyridostigmine, an acetylcholinesterase inhibitor that improves neuromuscular transmission, could improve NMJ function and thereby muscle strength and fatigability in patients with SMA.59%30061431
drug efficacySelf-reported reduction of fatigability and improved endurance shuttle test combined score performance suggest that pyridostigmine may be useful as an additional therapy to survival motor neuron-augmenting drugs (clinical trial result).59%36632180
drug efficacyPatients with SMA using pyridostigmine had fourfold smaller decreases in frequency and twofold smaller increases in amplitudes of sEMG signals in some muscles, recorded during ESTs (p < 0.05) (clinical trial result)51%37595479
safetyPatients with spinal muscular atrophy tolerated pyridostigmine well (clinical trial result).45%36632180

Off-Target Findings (255)

T1: 2T3: 230untiered: 23
TargetRoleTierBoltz-2 iPTMChai-1 iPTMVerdict
PAK4kinaseSMAT10.872Primary SMA hit
KCNA2ion_channelSMAT10.531Primary SMA hit
OPRK1gpcrOFF-TARGETT30.984Selectivity flag (off-target substrate)
TGFBR1kinaseSMAT30.975Selectivity flag (off-target substrate)
CHEK1kinaseOFF-TARGETT30.974Selectivity flag (off-target substrate)
BTKkinaseOFF-TARGETT30.971Selectivity flag (off-target substrate)
CHRM1gpcrOFF-TARGETT30.970Selectivity flag (off-target substrate)
GPR183gpcrOFF-TARGETT30.967Selectivity flag (off-target substrate)
CCKBRgpcrOFF-TARGETT30.964Selectivity flag (off-target substrate)
CSKkinaseOFF-TARGETT30.958Selectivity flag (off-target substrate)
DRD4gpcrOFF-TARGETT30.957Selectivity flag (off-target substrate)
AURKCkinaseOFF-TARGETT30.955Selectivity flag (off-target substrate)
HTR5AgpcrOFF-TARGETT30.954Selectivity flag (off-target substrate)
OPN1SWgpcrOFF-TARGETT30.954Selectivity flag (off-target substrate)
PDPK1kinaseOFF-TARGETT30.953Selectivity flag (off-target substrate)
RIPK2kinaseOFF-TARGETT30.953Selectivity flag (off-target substrate)
GPR135gpcrOFF-TARGETT30.953Selectivity flag (off-target substrate)
MAP2K3kinaseOFF-TARGETT30.952Selectivity flag (off-target substrate)
HRH3gpcrOFF-TARGETT30.952Selectivity flag (off-target substrate)
MAP3K3kinaseOFF-TARGETT30.952Selectivity flag (off-target substrate)
SSTR4gpcrOFF-TARGETT30.950Selectivity flag (off-target substrate)
FFAR2gpcrOFF-TARGETT30.950Selectivity flag (off-target substrate)
FFAR4gpcrOFF-TARGETT30.949Selectivity flag (off-target substrate)
GRK1kinaseOFF-TARGETT30.949Selectivity flag (off-target substrate)
S1PR4gpcrOFF-TARGETT30.948Selectivity flag (off-target substrate)
EPHA1kinaseOFF-TARGETT30.948Selectivity flag (off-target substrate)
GRK5kinaseOFF-TARGETT30.946Selectivity flag (off-target substrate)
ULK3kinaseOFF-TARGETT30.945Selectivity flag (off-target substrate)
DRD1gpcrOFF-TARGETT30.944Selectivity flag (off-target substrate)
CDK7kinaseOFF-TARGETT30.943Selectivity flag (off-target substrate)
AURKBkinaseOFF-TARGETT30.942Selectivity flag (off-target substrate)
GRK4kinaseOFF-TARGETT30.934Selectivity flag (off-target substrate)
HTR1EgpcrOFF-TARGETT30.932Selectivity flag (off-target substrate)
ADRA1DgpcrOFF-TARGETT30.931Selectivity flag (off-target substrate)
GIPRgpcrOFF-TARGETT30.929Selectivity flag (off-target substrate)
TNK1kinaseOFF-TARGETT30.929Selectivity flag (off-target substrate)
PAK2kinaseOFF-TARGETT30.922Selectivity flag (off-target substrate)
OPN5gpcrOFF-TARGETT30.920Selectivity flag (off-target substrate)
MAP3K13kinaseOFF-TARGETT30.917Selectivity flag (off-target substrate)
LTB4R2gpcrOFF-TARGETT30.916Selectivity flag (off-target substrate)
KSR1kinaseOFF-TARGETT30.914Selectivity flag (off-target substrate)
OPRL1gpcrOFF-TARGETT30.911Selectivity flag (off-target substrate)
BMXkinaseOFF-TARGETT30.910Selectivity flag (off-target substrate)
HCRTR2gpcrOFF-TARGETT30.910Selectivity flag (off-target substrate)
LPAR6gpcrOFF-TARGETT30.909Selectivity flag (off-target substrate)
HTR6gpcrOFF-TARGETT30.907Selectivity flag (off-target substrate)
GPR55gpcrOFF-TARGETT30.905Selectivity flag (off-target substrate)
HCAR3gpcrOFF-TARGETT30.902Selectivity flag (off-target substrate)
MAPK7kinaseOFF-TARGETT30.901Selectivity flag (off-target substrate)
MCHR1gpcrOFF-TARGETT30.899Selectivity flag (off-target substrate)

Showing top 50 of 255 targets.

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