Golodirsen: uses, dosing, side effects & brands
Golodirsen is a medicine sold in the U.S. under one brand, for duchenne muscular dystrophy. Below: what the FDA label says, every product that contains it, what the pills look like, and its recall record.
Key facts
- Treats
- Duchenne Muscular Dystrophy
- Available as
- Injectable
- Sold as
- Vyondys 53
- Prescription?
- Prescription only
- Generic available?
- Not in our catalog
How golodirsen is dosed
From the FDA label for Vyondys 53 (application NDA211970). Other golodirsen products — different forms, different strengths — are dosed differently. Follow the label for the one you were prescribed.
Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be measured before starting VYONDYS 53 ( 2.1 ) 30 milligrams per kilogram once weekly ( 2.2 ) Administer as an intravenous infusion over 35 to 60 minutes via an in-line 0.2 micron filter ( 2.2 , 2.4 ) Dilution required prior to administration ( 2.3 ) 2.1 Monitoring to Assess Safety Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be measured before starting VYONDYS 53. Consider measurement of glomerular filtration rate prior to initiation of VYONDYS 53. Monitoring for kidney toxicity during treatment is recommended. Obtain the urine samples prior to infusion of VYONDYS 53 or at least 48 hours after the most recent infusion [see Warnings and Precautions ( 5.2 )] . 2.2 Dosing Information The recommended dosage of VYONDYS 53 is 30 milligrams per kilogram administered once weekly as a 35 to 60-minute intravenous infusion via an in-line 0.2 micron filter. If a dose of VYONDYS 53 is missed, it may be administered as soon as possible after the scheduled dose. 2.3 Preparation Instructions VYONDYS 53 is supplied in single-dose vials as a preservative-free concentrated solution that requires dilution prior to administration. Parenteral drug products should be inspected visually for particulate matter and discoloration prior to administration, whenever solution and container…
Golodirsen side effects
The following serious adverse reactions are described below and elsewhere in the labeling: Hypersensitivity Reactions [see Warnings and Precautions ( 5.1 )] The most common adverse reactions (incidence ≥20% and higher than placebo) were headache, pyrexia, fall, abdominal pain, nasopharyngitis, cough, vomiting, and nausea. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Sarepta Therapeutics, Inc. at 1-888-SAREPTA (1-888-727-3782) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. In the VYONDYS 53 clinical development program, 58 patients received at least one intravenous dose of VYONDYS 53, ranging between 4 mg/kg (0.13 times the recommended dosage) and 30 mg/kg (the recommended dosage). All patients were male and had genetically confirmed Duchenne muscular dystrophy. Age at study entry was 6 to 13 years. Most (86%) patients were Caucasian. VYONDYS 53 was studied in 2 double-blind, placebo-controlled studies. In Study 1 Part 1, patients were randomized to receive once-weekly intravenous infusions of VYONDYS 53 (n=8) in four increasing dose levels from 4 mg/kg to 30 mg/kg or…
Who shouldn’t take golodirsen
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 [see Warnings and Precautions ( 5.1 )] . VYONDYS 53 is contraindicated in patients with serious hypersensitivity to golodirsen or to any of the inactive ingredients in VYONDYS 53. ( 4 , 5.1 )
Every golodirsen product we track (1)
Same active ingredient — different manufacturer, form, price and FDA recall record. That last one is what our independent score measures.
Only one: Vyondys 53.
What people report to the FDA about golodirsen
The FDA Adverse Event Reporting System (FAERS) collects reports from patients and clinicians. It holds 13 reports naming golodirsen, and the FDA flagged 77% of those reports as serious. The effects reported most often — leaving out reports about overdose, misuse or the condition being treated, which dominate the raw list for common medicines:
- cystatin c increased2 reports
- epilepsy2 reports
- abdominal pain1 reports
- acute respiratory failure1 reports
- atrioventricular block complete1 reports
- chest discomfort1 reports
- decreased appetite1 reports
- dehydration1 reports
Read these as a signal, not a rate. A report does not mean golodirsen caused the effect — anyone can file one, and many describe people taking several medicines for several conditions. Crucially there is no denominator: FAERS does not record how many people took the drug, so these counts cannot be turned into “X% of patients” — a bigger number often just means a more widely used or more talked-about drug. Duplicates exist, and publicity drives reporting. For what is actually established, read the FDA label section above.
Source: openFDA drug/event (FAERS), retrieved July 25, 2026.
Related calculators
Frequently asked questions
What is golodirsen?
VYONDYS 53 (golodirsen) injection is a sterile, aqueous, preservative-free, concentrated solution for dilution prior to intravenous administration. VYONDYS 53 is a clear to slightly opalescent, colorless liquid, and may contain trace amounts of small, white to off-white amorphous particles. VYONDYS 53 is supplied in single-dose vials containing 100 mg golodirsen (50 mg/mL). VYONDYS 53 is formulated as an isotonic phosphate buffered saline solution with an osmolality of 260 to 320 mOSM and a pH of 7.5. Each milliliter of VYONDYS 53 contains: 50 mg golodirsen; 0.2 mg potassium chloride; 0.2 mg potassium phosphate monobasic; 8 mg sodium chloride; and 1.14 mg sodium phosphate dibasic, anhydrous, in water for injection. The product may contain hydrochloric acid or sodium hydroxide to adjust pH. Golodirsen is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer (PMO) subclass. PMOs are synthetic molecules in which the five-membered ribofuranosyl rings found in natural DNA and RNA are replaced by a six-membered morpholino ring. Each morpholino ring is linked through an uncharged phosphorodiamidate moiety rather than the negatively charged phosphate linkage that is present in natural DNA and RNA. Each phosphorodiamidate morpholino subunit contains one of the heterocyclic bases found in DNA (adenine, cytosine, guanine, or thymine). Golodirsen contains 25 linked…
Can you take golodirsen with other medicines?
It depends on the medicine. We check it against the FDA labels rather than guessing: our interaction checker searches each drug's own label for the other and quotes what it says, naming the section it came from. Run golodirsen against whatever else you take — and remember that a label not naming a drug is not the same as that combination being safe.
What forms does golodirsen come in?
Across the brands we track, golodirsen is currently marketed as injectable, per the FDA's National Drug Code Directory. Each form is dosed differently — follow the label for the exact product you were prescribed.
Is there a generic golodirsen?
We do not currently list a generic-labelled golodirsen product. That does not always mean none exists — it means none appears under a generic name in the FDA data we track. Ask your pharmacist.
Cite this page
- APA
- pharmaranks. (2026, July 24). Golodirsen: uses, dosing, side effects & brands. https://pharmaranks.com/drugs/golodirsen
- MLA
- “Golodirsen: uses, dosing, side effects & brands.” pharmaranks, 24 July 2026, https://pharmaranks.com/drugs/golodirsen.
We summarise public FDA and NIH sources — for a clinical claim, cite the primary source we link to as well.
Sources: FDA openFDA drug label, National Drug Code Directory, and Enforcement (recall) database. This page reproduces public FDA data and is not medical advice. Dosing is set by your prescriber.
Read the full FDA label for golodirsen on DailyMed (NIH) ↗.