Golodirsen
Golodirsen is an antisense oligonucleotide therapy designed to treat Duchenne muscular dystrophy (DMD) in patients whose genetic mutations are amenable to exon 53 skipping. It works by binding to dystrophin pre-mRNA and forcing exclusion of exon 53 during mRNA processing, allowing production of a shortened but functional dystrophin protein that helps stabilize muscle cells.
Golodirsen is an exon-skipping therapy for Duchenne muscular dystrophy that enables production of a truncated but functional dystrophin protein.
Key facts at a glance
- ATC
- M09AX08
- RxNorm
- 2267207
- Generic
- Golodirsen
- Class
- atc-m09
- Brands
- Vyondys 53
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- injection
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Dosage and administration
- Usual dose
- 30mg/kg once weekly
- Maximum
- consult physician
- Frequency
- once weekly
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 30mg/kg once weekly
- Maximum
- consult physician
- Frequency
- once weekly
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 30mg/kg once weekly
- Maximum
- consult physician
- Frequency
- once weekly
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
Overdose information
Special Populations
10 groupspregnancy
generalavoid if possible
avoid use during pregnancy unless benefits outweigh risks
regular fetal monitoring
pregnancy
first trimesteravoid if possible
avoid use during first trimester unless benefits outweigh risks
regular fetal monitoring and ultrasound assessments
lactation
generaldiscontinue or avoid
breastfeeding not recommended during treatment
monitor infant for adverse effects
pediatric_age
age 5 to 18no adjustment required
indicated for patients with DMD amenable to exon 53 skipping; dosing based on weight
regular monitoring of renal function and infusion reactions
pediatric_age
under 5use with caution
safety and efficacy not established in patients under 5 years
enhanced monitoring required if used
geriatric
age 65 and aboveuse with caution
limited data in patients over 65; DMD is primarily pediatric
monitor renal and hepatic function
renal_impairment
mild to moderatemonitor closely
monitor renal function regularly; no specific dose adjustment recommended
regular monitoring of serum creatinine and glomerular filtration rate
Use with caution and monitor for adverse effects.
renal_impairment
severeuse with caution
limited data in severe renal impairment; use only if benefits outweigh risks
frequent renal function monitoring required
Use with caution and monitor for adverse effects.
hepatic_impairment
milduse with caution
limited data in hepatic impairment
monitor liver function tests
hepatic_impairment
moderate to severeuse with caution
not studied in moderate to severe hepatic impairment
monitor liver function tests
Statistics and clinical data
FDA approval year
FDA approval date for Golodirsen (Vyondys 53)
Drug class
Pharmacological classification of Golodirsen
Therapeutic indication
Primary therapeutic use
Golodirsen is indicated for the treatment of Duchenne muscular dystrophy in patients with confirmed genetic mutations amenable to exon 53 skipping. Genetic testing is required to determine eligibility.
Golodirsen is an antisense oligonucleotide that specifically binds to exon 53 of the dystrophin pre-mRNA. This binding causes the exon to be excluded during mRNA processing, resulting in production of an internally truncated but functionally active dystrophin protein.
Administered as weekly intravenous infusions. Common adverse reactions include infusion reactions and headache. Kidney function monitoring is recommended. May increase risk of respiratory infections. Not effective in DMD patients without exon 53 amenable mutations.
Contraindications
Conditions and patient states where this medication should be avoided or used with caution.
Do not use (1)
Known allergy to this medication or its components
Use caution (1)
Consult healthcare provider for benefits vs risks assessment
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy