Substance / Medication

Risdiplam

Overview

Active Ingredient
risdiplam
RxNorm CUI
2390935

Indications

EVRYSDI is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.

Labeler: Genentech, Inc.Updated: 2026-02-19T00:00:00.000ZFull label on DailyMed

Contraindications

When this intervention should not be used

None.

Indications & Related Conditions

Conditions associated via SNOMED clinical relationships

Administration & Protocol

Dosing, route, and treatment protocol

Detailed dosage and administration information is available in the full FDA drug label.

View full prescribing information on DailyMed

Monitoring & Follow-Up

Biomarkers relevant to this intervention via related conditions

No monitoring biomarkers have been mapped yet. Biomarker-intervention linkages are derived through related conditions and will expand as the knowledge graph grows.

Research Evidence

Published studies and systematic reviews

Sort:
Safety and effectiveness of risdiplam in adults with spinal muscular atrophy: a systematic review.
Alonge Paolo, Urbano Guido, Gadaleta Giulio · J Neurol · 2025
PMID: 41354988Meta-AnalysisFull text (PMC)
Efficacy of risdiplam in spinal muscular atrophy: A systematic review and meta-analysis.
Pascual-Morena Carlos, Martínez-Vizcaíno Vicente, Cavero-Redondo Iván et al. · Pharmacotherapy · 2024
PMID: 37574770Meta-Analysis
The Impact of Nusinersen and Risdiplam on Motor Function for Spinal Muscular Atrophy Type 2 and 3: A Meta-Analysis.
Chen Bo, Gong Yuanlin, Zhou Tengteng · J Coll Physicians Surg Pak · 2024
PMID: 39113515Meta-Analysis
How does risdiplam compare with other treatments for Types 1-3 spinal muscular atrophy: a systematic literature review and indirect treatment comparison.
Ribero Valerie Aponte, Daigl Monica, Martí Yasmina et al. · J Comp Eff Res · 2022
PMID: 35040693Meta-Analysis
Risdiplam in types 2 and 3 spinal muscular atrophy: A randomised, placebo-controlled, dose-finding trial followed by 24 months of treatment.
Mercuri Eugenio, Baranello Giovanni, Boespflug-Tanguy Odile et al. · Eur J Neurol · 2023
PMID: 35837793RCT
Matched-pair analysis of motor outcomes in adults with spinal muscular atrophy on nusinersen vs. risdiplam.
Svenja Neuhoff, Benjamin Stolte, Jaqueline Lipka et al. · J Neurol · 2026
PMID: 41484582ObservationalFull text (PMC)

Research data from MEDLINE/PubMed

Benefits & Expected Outcomes

Benefits, expected outcomes, efficacy data, and NNT (Number Needed to Treat) are pending physician authorship and evidence review.

Risks & Side Effects

Adverse reaction and safety data for this drug is sourced from the FDA-approved label.

View adverse reactions & drug interactions on DailyMed

Related Symptoms

Symptoms associated with conditions this intervention addresses

No related symptoms have been mapped yet. Symptom linkages are derived through associated conditions.

Alternatives & Comparisons

Alternative treatments, comparison data, and clinical decision support are pending physician authorship.

Medical Disclaimer

This information is for educational purposes only and does not constitute medical advice, diagnosis, or treatment. Always consult with a qualified healthcare provider regarding any medical condition or treatment plan.

Do not start, stop, or change any treatment without consulting your healthcare provider.

Quick Facts

Type
Substance / Medication
Fully Specified Name
Risdiplam (substance)
SNOMED CT
896813005
UMLS CUI
C5139676
RxNorm CUI
2390935
Labeler
Genentech, Inc.

Clinical Data

This intervention maps to 1 entities in the Ltrl knowledge graph.

1
Conditions
0
Biomarkers
0
Specialists
0
Symptoms

Data is sourced from SNOMED CT, UMLS, and the Ltrl clinical knowledge graph. Content sections marked as pending require physician authorship. Consult a healthcare provider before starting any treatment.