SMA Research Platform

Evidence graph for Spinal Muscular Atrophy

Biology-first target discovery
Christian Fischer / Bryzant Labs
15,934Targets
453Trials
84Drugs
7Datasets
7,947Sources
226,991Claims
263,546Evidence
58,010Hypotheses
DRUGsmall molecule

4AP

Related claims (4)

TypePredicateConfSource
motor function4-aminopyridine (4AP) can significantly increase twitch force and EMG of failing motor units in HCSMA homozygotes (observed in animal model).50%PMID:9151766
motor function4-aminopyridine (4AP) increases peak forces during motor unit tetanic activation but does not eliminate tetanic force failure in HCSMA homozygotes (observed in animal model).50%PMID:9151766
drug efficacyThe ability of 4-aminopyridine (4AP) to increase force in failing motor units may be related to the extent of motor unit failure (observed in animal model).42%PMID:9151766
drug efficacy4-aminopyridine (4AP) increases nerve-evoked whole muscle twitch force and electromyograms (EMG) to a greater extent in older homozygous HCSMA animals than in similarly aged, symptomless HCSMA animals or younger homozygous animals (observed...42%PMID:9151766

Off-Target Findings (29)

T4: 1untiered: 28
TargetRoleTierBoltz-2 iPTMChai-1 iPTMVerdict
CHRNA1_ECDOTHERT40.938Low relevance
RIPK3_kinaseUNCLASSIFIED0.976
LIMK2_aCOTHER0.961
PLS3_EF4OTHER0.876
KCNA2_poreOTHER0.874
SMN1_YGboxOTHER0.864
AQP4_M1UNCLASSIFIED0.863
CFL2SMA0.840
LIMK1_aCOTHER0.824
PFN2SMA0.815
STMN1SMA0.767
ROCK2_aCOTHER0.763
EIF4E_capbindingUNCLASSIFIED0.747
S100B_dimerUNCLASSIFIED0.741
PERP_ECL1OTHER0.740
DNMT3B_catalyticUNCLASSIFIED0.730
AGRN_LG3OTHER0.722
GAP43SMA0.697
HSP70_NBDOTHER0.686
RAPSN_ntermOTHER0.666
LRP4_propellerOTHER0.653
RIPK1_kinaseUNCLASSIFIED0.599
ROCK1_aCOTHER0.564
MLKL_4HBUNCLASSIFIED0.552
NCALDSMA0.547
CFL1SMA0.527
CLCN1_poreUNCLASSIFIED0.500
CHRNG_fetalOTHER0.495
DOK7_PHOTHER0.468
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