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
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 (19)

T4: 1untiered: 18
TargetRoleTierBoltz-2 iPTMChai-1 iPTMVerdict
CHRNA1_ECDOTHERT40.933Low relevance
LIMK2_aCOTHER0.961
KCNA2_poreOTHER0.868
CFL2SMA0.840
PFN2SMA0.815
PLS3_EF4OTHER0.782
STMN1SMA0.764
AGRN_LG3OTHER0.722
GAP43SMA0.697
PERP_ECL1OTHER0.684
RAPSN_ntermOTHER0.666
HSP70_NBDOTHER0.645
ROCK2_aCOTHER0.607
LRP4_propellerOTHER0.587
SMN1_YGboxOTHER0.549
NCALDSMA0.547
ROCK1_aCOTHER0.480
CFL1SMA0.444
DOK7_PHOTHER0.405
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