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GAA

Lysosomal alpha-glucosidase · P10253 · GAA on Sugi Atlas →

2,874,831 patent compounds predicted against GAA, 1,558,969 high-confidence (nearest known ligand ≥ 0.5)

Top predicted compounds (top 20 by supporting neighbours, then confidence)

CompoundConfidenceSupporting neighbours
SCHEMBL8112 SCHEMBL8112 0.47 11/20
SCHEMBL4651 SCHEMBL4651 0.59 7/20
Duvoglustat Duvoglustat (SCHEMBL2969) 1.00 6/20
SCHEMBL5938 SCHEMBL5938 0.55 6/20
Esculin Esculin (SCHEMBL2893) 1.00 5/20
Esculin Esculin (SCHEMBL2894) 1.00 5/20
Teniposide Teniposide (SCHEMBL3907) 1.00 5/20
Teniposide Teniposide (SCHEMBL3908) 1.00 5/20
Etoposide Etoposide (SCHEMBL4258) 1.00 5/20
Etoposide Etoposide (SCHEMBL4259) 1.00 5/20
Etoposide Etoposide (SCHEMBL3622) 0.94 5/20
Etocarlide Etocarlide (SCHEMBL676) 0.83 5/20
Etocarlide Etocarlide (SCHEMBL3292) 0.83 5/20
Etoposide Phosphate Etoposide Phosphate (SCHEMBL3621) 0.83 5/20
SCHEMBL6710 SCHEMBL6710 0.64 5/20
Prometryn Prometryn (SCHEMBL7622) 0.54 5/20
SCHEMBL1862 SCHEMBL1862 0.56 4/20
SCHEMBL1863 SCHEMBL1863 0.56 4/20
SCHEMBL6181 SCHEMBL6181 0.52 4/20
SCHEMBL572 SCHEMBL572 0.52 4/20

Clinical trials — most relevant to GAA by text similarity (a relevance ranking, not an exhaustive list)

TrialPhaseStatus
Growth and Development Study of Alglucosidase Alfa PHASE4COMPLETED
Pharmacokinetics, Pharmacodynamics And Safety Study Of Elelyso(tm) In Pediatric Subjects With Type 1 Gaucher Disease PHASE4WITHDRAWN
High Dose or High Dose Frequency Study of Alglucosidase Alfa PHASE4COMPLETED
Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa PHASE3ACTIVE_NOT_RECRUITING
A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease PHASE3COMPLETED
Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support PHASE3COMPLETED
A Study of rhGAA in Patients With Late-Onset Pompe Disease PHASE2COMPLETED
High-Dose Ambroxol in GBA1-Related Parkinson PHASE1/PHASE2UNKNOWN
Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease PHASE2COMPLETED
rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease) PHASE1/PHASE2COMPLETED
A Phase I Study of Pyrimethamine in Patients With GM2 Gangliosidosis PHASE1WITHDRAWN
Biomarker for Glycogen Storage Diseases (BioGlycogen) nanWITHDRAWN
Cardiopulmonary Exercise Test to Quantify Enzyme Replacement Response in Pediatric Pompe Disease nanCOMPLETED
Detection of Pompe Disease in Adult Patients With Myopathies of Uncertain Origin or With Asymptomatic Hyper-CK-emia nanUNKNOWN
Screening Protocol to Evaluate Acid Alpha-Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease nanCOMPLETED
Assessment of Neurological Manifestations in Gaucher Disease Patients nanUNKNOWN
Alglucosidase Alfa Temporary Access Program nanAPPROVED_FOR_MARKETING
A Study to Evaluate and Characterize the Effect of Pharmacological Chemicals on Blood From Patients With Gaucher Disease nanCOMPLETED
Registry Gangliosidoses nanUNKNOWN
Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-onset Pompe Disease nanAPPROVED_FOR_MARKETING
Lyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher Disease nanCOMPLETED
Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe Disease nanCOMPLETED
Pregnancy and Birth Outcome in Women With Pompe Disease nanCOMPLETED
Study to Collect Data on Fabry Disease Patients With Enhanceable Alpha-Galactosidase A Activity nanCOMPLETED
MSOT in Pompe Disease nanCOMPLETED
Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease nanRECRUITING
The Efficacy and Safety of Switch Between Agalsidase Beta to Agalsidase Alfa for Enzyme Replacement in Patients With Anderson-Fabry Disease nanCOMPLETED
Newborn Screening Assay of Pompe's Disease nanCOMPLETED
Exercise Capacity in Response to Enzyme Replacement Therapy in Pediatric Pompe Disease. nanUNKNOWN
Effect of Migalastat on Cardiac Involvement in Fabry Disease nanCOMPLETED

Related proteins — ESM-2 sequence neighbours (a GAA binder may also engage these)

ProteinNameSimilarity
P70699 — 1.000 P70699 →
Q9MYM4 — 1.000 Q9MYM4 →
Q5R7A9 — 1.000 Q5R7A9 →
Q6P7A9 — 0.997 Q6P7A9 →
Q8BHN3 — 0.994 Q8BHN3 →
GANAB Neutral alpha-glucosidase AB 0.992 landscape →
Q4R4N7 — 0.991 Q4R4N7 →
P79403 — 0.991 P79403 →
Q5RDJ3 — 0.986 Q5RDJ3 →
P19813 — 0.985 P19813 →
O35632 — 0.985 O35632 →
O54782 — 0.984 O54782 →
Q91ZJ9 — 0.983 Q91ZJ9 →
Q69ZQ1 — 0.983 Q69ZQ1 →
P17164 — 0.983 P17164 →
Q58DH9 — 0.982 Q58DH9 →
O04931 — 0.981 O04931 →
Q8SQG8 — 0.981 Q8SQG8 →
Q3U4H6 — 0.981 Q3U4H6 →
DPEP1 Dipeptidase 1 0.981 landscape →
Q9Y6R7 — 0.981 Q9Y6R7 →
Q60HF8 — 0.981 Q60HF8 →
MGAM2 Probable maltase-glucoamylase 2 (Maltase-gluc 0.981 landscape →
O97524 — 0.981 O97524 →
P51569 — 0.981 P51569 →
Q12891 — 0.980 Q12891 →
Q3SZM7 — 0.980 Q3SZM7 →
Q43763 — 0.980 Q43763 →
Q9UQQ1 — 0.980 Q9UQQ1 →
MAN2B2 Epididymis-specific alpha-mannosidase 0.980 landscape →