risdiplam
E133682
Risdiplam is an oral small-molecule drug used to treat spinal muscular atrophy by increasing production of functional survival motor neuron (SMN) protein.
All labels observed (1)
| Label | Occurrences |
|---|---|
| risdiplam canonical | 2 |
How this entity was disambiguated
This entity first appeared as the object of triple T1169483 — 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.
Target entity: risdiplam Context triple: [spinal muscular atrophy, treatedBy, risdiplam]
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A.
Sikorski–Mayski agreement
The Sikorski–Mayski agreement was a 1941 pact between the Polish government-in-exile and the Soviet Union that restored diplomatic relations and led to an "amnesty" for many Polish citizens imprisoned or deported in the USSR during World War II.
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B.
Linas-Marcoussis Agreement
The Linas-Marcoussis Agreement is a 2003 French-brokered peace accord aimed at ending the civil conflict in Côte d’Ivoire by establishing a power-sharing government and political reforms.
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C.
Einigungsvertrag
Der Einigungsvertrag ist der Staatsvertrag von 1990, der die Bedingungen und rechtlichen Grundlagen der deutschen Wiedervereinigung zwischen der Bundesrepublik Deutschland und der DDR festlegte.
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D.
Madrid Accords
The Madrid Accords were a 1975 agreement in which Spain agreed to withdraw from Spanish Sahara and transfer its administration to Morocco and Mauritania, paving the way for the territory’s contested annexation.
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E.
A Revision of the Treaty
A Revision of the Treaty is John Maynard Keynes’s 1922 follow-up work to The Economic Consequences of the Peace, in which he further critiques and proposes modifications to the post–World War I peace settlement.
- F. None of above. chosen
- G. Unsure - the case is ambiguous/there is not enough information to decide.
Target entity: risdiplam Target entity description: Risdiplam is an oral small-molecule drug used to treat spinal muscular atrophy by increasing production of functional survival motor neuron (SMN) protein.
-
A.
Sikorski–Mayski agreement
The Sikorski–Mayski agreement was a 1941 pact between the Polish government-in-exile and the Soviet Union that restored diplomatic relations and led to an "amnesty" for many Polish citizens imprisoned or deported in the USSR during World War II.
-
B.
Linas-Marcoussis Agreement
The Linas-Marcoussis Agreement is a 2003 French-brokered peace accord aimed at ending the civil conflict in Côte d’Ivoire by establishing a power-sharing government and political reforms.
-
C.
Einigungsvertrag
Der Einigungsvertrag ist der Staatsvertrag von 1990, der die Bedingungen und rechtlichen Grundlagen der deutschen Wiedervereinigung zwischen der Bundesrepublik Deutschland und der DDR festlegte.
-
D.
Madrid Accords
The Madrid Accords were a 1975 agreement in which Spain agreed to withdraw from Spanish Sahara and transfer its administration to Morocco and Mauritania, paving the way for the territory’s contested annexation.
-
E.
A Revision of the Treaty
A Revision of the Treaty is John Maynard Keynes’s 1922 follow-up work to The Economic Consequences of the Peace, in which he further critiques and proposes modifications to the post–World War I peace settlement.
- F. None of above. chosen
Statements (49)
| Predicate | Object |
|---|---|
| instanceOf |
disease-modifying therapy for spinal muscular atrophy
ⓘ
orally administered medication ⓘ small-molecule drug ⓘ splicing modifier ⓘ |
| actsOnGene | SMN2 ⓘ |
| administeredOnce | daily ⓘ |
| administrationPopulation |
adults
ⓘ
children ⓘ infants ⓘ |
| administrationWithFood | can be taken with or without food ⓘ |
| affectsProtein |
survival motor neuron protein
ⓘ
surface form:
SMN protein
|
| approvedBy |
European Medicines Agency
ⓘ
Food and Drug Administration ⓘ
surface form:
U.S. Food and Drug Administration
|
| clinicalTrialProgram |
FIREFISH
ⓘ
JEWELFISH ⓘ RAINBOWFISH ⓘ SUNFISH ⓘ |
| commonAdverseEvent |
diarrhea
ⓘ
fever ⓘ rash ⓘ upper respiratory tract infection ⓘ |
| contraindication | known hypersensitivity to risdiplam or its excipients ⓘ |
| developedBy |
PTC Therapeutics
ⓘ
Roche ⓘ |
| developedInCollaborationWith | SMA Foundation ⓘ |
| dosageForm | oral solution ⓘ |
| eliminationRoute | mainly via feces ⓘ |
| hasATCCode | M09AX10 ⓘ |
| hasBrandName | Evrysdi ⓘ |
| hasMolecularTarget |
SMN2
ⓘ
surface form:
SMN2 pre-mRNA
|
| improvesClinicalOutcome | motor function in patients with spinal muscular atrophy ⓘ |
| increasesProductionOf | functional SMN protein ⓘ |
| indicatedFor | 5q-associated spinal muscular atrophy ⓘ |
| indication |
treatment of spinal muscular atrophy in adult patients
ⓘ
treatment of spinal muscular atrophy in pediatric patients ⓘ |
| mechanismOfAction | modulation of SMN2 pre-mRNA splicing to promote exon 7 inclusion ⓘ |
| metabolism | primarily hepatic ⓘ |
| pharmacologicalClass |
SMN2 splicing modifier
ⓘ
survival motor neuron protein upregulator ⓘ |
| pregnancyCategory | may cause fetal harm based on animal data ⓘ |
| regulatoryDesignation | orphan drug for spinal muscular atrophy ⓘ |
| regulatoryStatus | prescription-only medicine ⓘ |
| requiresMonitoringOf |
liver function
ⓘ
ophthalmologic status in some patients ⓘ |
| routeOfAdministration | oral ⓘ |
| storageCondition | refrigeration before reconstitution (for some formulations) ⓘ |
| targetsBiologicalProcess | pre-mRNA splicing of SMN2 ⓘ |
| therapeuticEffect | increase in SMN protein levels in blood and tissues ⓘ |
| treats | spinal muscular atrophy ⓘ |
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.
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.
Subject: risdiplam Description of subject: Risdiplam is an oral small-molecule drug used to treat spinal muscular atrophy by increasing production of functional survival motor neuron (SMN) protein.
Referenced by (2)
Full triples — surface form annotated when it differs from this entity's canonical label.