onasemnogene abeparvovec
E133681
Onasemnogene abeparvovec is a gene therapy medication that delivers a functional copy of the SMN1 gene to treat infants and young children with certain forms of spinal muscular atrophy.
All labels observed (2)
| Label | Occurrences |
|---|---|
| Zolgensma | 5 |
| onasemnogene abeparvovec canonical | 5 |
How this entity was disambiguated
This entity first appeared as the object of triple T1169482 — resolving that mention is where its identity was fixed. The disambiguator weighed these candidate entities and picked the highlighted one (or “None”, minting a new entity). This is how homonymy is resolved: the same surface form can point to different entities.
NED1
Entity disambiguation (via context triple)
gpt-5-mini-2025-08-07
Target entity: onasemnogene abeparvovec Context triple: [spinal muscular atrophy, treatedBy, onasemnogene abeparvovec]
-
A.
Spinraza
Spinraza is a prescription medication used to treat spinal muscular atrophy (SMA) by modifying SMN2 gene expression to increase production of survival motor neuron (SMN) protein.
-
B.
Aduhelm
Aduhelm is an Alzheimer’s disease drug developed by Biogen that targets amyloid-beta plaques in the brain.
-
C.
Leqembi
Leqembi is an Alzheimer’s disease drug (lecanemab) that targets amyloid-beta plaques to slow cognitive decline in early-stage patients.
-
D.
Anpezan
Anpezan is a regional dialect of the Ladin language spoken in parts of the Dolomite area of northern Italy.
-
E.
Tysabri
Tysabri is a monoclonal antibody medication primarily used to treat relapsing forms of multiple sclerosis and Crohn’s disease.
- F. None of above. chosen
- G. Unsure - the case is ambiguous/there is not enough information to decide.
NED2
Entity disambiguation (via description)
gpt-5-mini-2025-08-07
Target entity: onasemnogene abeparvovec Target entity description: Onasemnogene abeparvovec is a gene therapy medication that delivers a functional copy of the SMN1 gene to treat infants and young children with certain forms of spinal muscular atrophy.
-
A.
Spinraza
Spinraza is a prescription medication used to treat spinal muscular atrophy (SMA) by modifying SMN2 gene expression to increase production of survival motor neuron (SMN) protein.
-
B.
Aduhelm
Aduhelm is an Alzheimer’s disease drug developed by Biogen that targets amyloid-beta plaques in the brain.
-
C.
Leqembi
Leqembi is an Alzheimer’s disease drug (lecanemab) that targets amyloid-beta plaques to slow cognitive decline in early-stage patients.
-
D.
Anpezan
Anpezan is a regional dialect of the Ladin language spoken in parts of the Dolomite area of northern Italy.
-
E.
Tysabri
Tysabri is a monoclonal antibody medication primarily used to treat relapsing forms of multiple sclerosis and Crohn’s disease.
- F. None of above. chosen
Statements (49)
| Predicate | Object |
|---|---|
| instanceOf |
AAV9-based gene therapy
ⓘ
biological medicinal product ⓘ gene therapy medication ⓘ orphan drug ⓘ |
| approvedBy |
European Medicines Agency
ⓘ
U.S. Food and Drug Administration ⓘ
linked to:
Food and Drug Administration
|
| approvedIn |
Canada
ⓘ
European Union ⓘ Japan ⓘ United States ⓘ
linked to:
United States of America
|
| coAdministeredWith | systemic corticosteroids ⓘ |
| commonAdverseEffect |
elevated liver enzymes
ⓘ
increased troponin-I ⓘ thrombocytopenia ⓘ vomiting ⓘ |
| delivers | functional copy of SMN1 gene ⓘ |
| developedBy |
AveXis
ⓘ
Novartis ⓘ |
| dosingRegimen | single-dose treatment ⓘ |
| EMAAuthorizationDate | 2020-05-18 ⓘ |
| FDAApprovalDate | 2019-05-24 ⓘ |
| hasAdministrationAgeLimit | less than 2 years (initial FDA label) ⓘ |
| hasATCCode | M09AX09 ⓘ |
| hasBlackBoxWarning | acute serious liver injury ⓘ |
| hasInternationalNonproprietaryName | onasemnogene abeparvovec ⓘ |
| hasListPriceUS | approximately 2.1 million USD per treatment at launch ⓘ |
| hasMechanismOfAction |
gene replacement therapy
ⓘ
increases SMN protein expression ⓘ |
| hasPregnancyCategory | not assigned ⓘ |
| hasStorageCondition | frozen ⓘ |
| hasTherapeuticArea |
neuromuscular disease
ⓘ
rare disease ⓘ |
| hasTradeName |
Zolgensma
ⓘ
linked to:
onasemnogene abeparvovec
|
| indicatedFor |
infants with spinal muscular atrophy
ⓘ
patients with biallelic mutations in the SMN1 gene ⓘ patients with three or fewer copies of the SMN2 gene ⓘ pediatric patients with spinal muscular atrophy ⓘ presymptomatic infants with spinal muscular atrophy ⓘ |
| isFirstInClass | yes ⓘ |
| legalStatusInEU | prescription-only medicine ⓘ |
| legalStatusInUS | prescription-only medicine ⓘ |
| requiresMonitoring |
cardiac troponin-I
ⓘ
liver function tests ⓘ platelet counts ⓘ |
| routeOfAdministration | intravenous infusion ⓘ |
| targetsGene |
SMN1
ⓘ
linked to:
SMN1 gene
|
| treats |
5q-associated spinal muscular atrophy
ⓘ
spinal muscular atrophy ⓘ |
| usesVector | adeno-associated virus serotype 9 ⓘ |
How these facts were elicited
The pipeline generated the facts above by prompting gpt-5.1 with this entity's name + description and the instruction below.
Instruction
You are a knowledge base construction expert. Given a subject entity and a description of it, return factual statements that you know for the subject as a JSON list of dictionaries(triples), where keys must be "subject", "predicate" and "object". The number of facts may be very high, between 25 to 50 or more, for very popular subjects. For less popular subjects, the number of facts can be very low, like 5 or 10. # Requirements - If you don't know the subject at all, return an empty list. - If the subject is not a named entity, return an empty list. - Include at least one triple where predicate is "instanceOf". - Do not get too wordy. - Separate several objects into multiple triples with one object.
Input
Subject: onasemnogene abeparvovec Description of subject: Onasemnogene abeparvovec is a gene therapy medication that delivers a functional copy of the SMN1 gene to treat infants and young children with certain forms of spinal muscular atrophy.
Referenced by (10)
Full triples — surface form annotated when it differs from this entity's canonical label.