RAINBOWFISH

E561099

RAINBOWFISH is a clinical trial evaluating risdiplam in infants with spinal muscular atrophy, particularly those pre-symptomatic or very young at treatment initiation.

All labels observed (1)

Label Occurrences
RAINBOWFISH canonical 1

How this entity was disambiguated

Statements (30)

Predicate Object
instanceOf clinical trial ⓘ
interventional study ⓘ
aimsTo treat spinal muscular atrophy before onset of clinical symptoms ⓘ
drugMechanismStudied increasing SMN protein levels via SMN2 splicing modification ⓘ
enrollmentCriteriaIncludes genetically confirmed spinal muscular atrophy ⓘ
very young age at first dose ⓘ
evaluates risdiplam ⓘ
focusesOn pre-symptomatic infants with spinal muscular atrophy ⓘ
very young infants at treatment initiation ⓘ
hasIntervention risdiplam ⓘ
hasPopulation infants with spinal muscular atrophy ⓘ
pre-symptomatic spinal muscular atrophy patients ⓘ
very young spinal muscular atrophy patients ⓘ
hasPrimaryObjective to assess efficacy of risdiplam in preventing or delaying symptoms of spinal muscular atrophy in infants ⓘ
hasSecondaryObjective to assess safety and tolerability of risdiplam in infants with spinal muscular atrophy ⓘ
investigates efficacy of risdiplam in spinal muscular atrophy ⓘ
safety of risdiplam in spinal muscular atrophy ⓘ
phase Phase II ⓘ
relatedTo Evrysdi clinical development program ⓘ
treatment of spinal muscular atrophy type 1 ⓘ
routeOfAdministration oral risdiplam ⓘ
sponsor Genentech ⓘ
Roche ⓘ
studiesCondition spinal muscular atrophy ⓘ
studiesDrug risdiplam (Evrysdi) ⓘ
linked to: Evrysdi
targetsAgeGroup infants ⓘ
targetsDiseaseStage pre-symptomatic stage of spinal muscular atrophy ⓘ
therapeuticArea neuromuscular disease ⓘ
rare disease ⓘ
usesDrugClass SMN2 splicing modifier ⓘ

How these facts were elicited

Referenced by (1)

Full triples — surface form annotated when it differs from this entity's canonical label.

risdiplam → clinicalTrialProgram → RAINBOWFISH ⓘ