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.
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 NERFINISHED ⓘ |
| focusesOn |
pre-symptomatic infants with spinal muscular atrophy
ⓘ
very young infants at treatment initiation ⓘ |
| hasIntervention | risdiplam NERFINISHED ⓘ |
| 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
NERFINISHED
ⓘ
Roche NERFINISHED ⓘ |
| studiesCondition | spinal muscular atrophy NERFINISHED ⓘ |
| studiesDrug | risdiplam (Evrysdi) NERFINISHED ⓘ |
| targetsAgeGroup | infants ⓘ |
| targetsDiseaseStage | pre-symptomatic stage of spinal muscular atrophy ⓘ |
| therapeuticArea |
neuromuscular disease
ⓘ
rare disease ⓘ |
| usesDrugClass | SMN2 splicing modifier ⓘ |
Referenced by (1)
Full triples — surface form annotated when it differs from this entity's canonical label.