Overview
Indications
DMD 14 [see Clinical Studies ()] VYONDYS 53 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of thegene that is amenable to exon 53 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VYONDYS 53. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.
Contraindications
When this intervention should not be used
5.1 [see Warnings and Precautions ()] VYONDYS 53 is contraindicated in patients with a serious hypersensitivity reaction to golodirsen or to any of the inactive ingredients in VYONDYS 53. Anaphylaxis has occurred in patients receiving VYONDYS 53.
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 DailyMedMonitoring & 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
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 DailyMedRelated 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.
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
- Golodirsen (substance)
- SNOMED CT
- 838470000
- UMLS CUI
- C5222004
- RxNorm CUI
- 2267207
- Labeler
- Sarepta Therapeutics, Inc.
Clinical Data
This intervention maps to 2 entities in the Healos knowledge graph.
Data is sourced from SNOMED CT, UMLS, and the Healos clinical knowledge graph. Content sections marked as pending require physician authorship. Consult a healthcare provider before starting any treatment.