How is Vyondys 53 administered?
Vyondys 53 (golodirsen) is given by intravenous (IV) infusion, meaning it is delivered directly into a vein through a needle by a healthcare professional.
Dosage and Administration
Vyondys 53 is given by a healthcare professional once a week.
- Infusion time: 35 to 60 minutes
- Filter requirement: Administered through an in-line 0.2-micron filter
- Dose: 30 mg per kg of body weight
- Venous access: Requires an indwelling venous access catheter
Infusion Process
- Preparation: A topical anesthetic cream may be applied to numb the injection site if needed.
- Pre-Infusion Flush: The IV access line is flushed with 0.9% Sodium Chloride Injection, USP before administering Vyondys 53.
- Infusion: The medication is delivered via IV over the recommended timeframe.
- Post-Infusion Flush: The IV access line is flushed again after the infusion is complete.
Potential Reactions
Hypersensitivity reactions can occur when Vyondys 53 is administered. If this happens then the infusion may need to be slowed or interrupted by a healthcare provider.
What is Vyondys 53 used for?
Vyondys is a medication used to treat Duchenne muscular dystrophy (DMD) in children and adults who have a mutation of the DMD gene that is amenable to exon 53 skipping.
This is not all the information you need to know about Vyondys 53 (golodirsen) for safe and effective use and does not take the place of your doctor’s directions. Review the full product information and discuss this information and any questions you have with your doctor or other health care provider.
References
- Food and Drug Administration (FDA). Vyondys 53. [Accessed October 13, 20201]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/211970s002lbl.pdf.
Read next
What are the new drugs for DMD (Duchenne muscular dystrophy)?
The new drugs approved by the FDA for the treatment of Duchenne Muscular Dystrophy (DMD) are Agamree, Amondys 45, Duvyzat, Elevidys, Emflaza, Exondys 51, Viltepso, and Vyondys 53. They include classes such as antisense oligonucleotides, glucocorticoid (corticosteroids), a gene therapy and a histone deacetylase (HDAC) inhibitor. Continue reading
How does Vyondys 53 work?
Vyondys 53 is an antisense oligonucleotide that works by binding to exon 53 of dystrophin pre-mRNA, which results in this exon being skipped over during mRNA processing. Skipping of exon 53 allows cells to join different exons together to produce a dystrophin protein that is shorter but has some function. Continue reading
How long does it take for Vyondys 53 to work?
Results from a small phase I/II clinical trial show that treatment with Vyondys 53 increases dystrophin protein levels within 48 weeks of starting treatment. Continue reading
Related medical questions
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Drug information
- Vyondys 53 Information for Consumers
- Vyondys 53 prescribing info & package insert (for Health Professionals)
- Side Effects of Vyondys 53 (detailed)
Related support groups
- Vyondys 53 (4 questions, 3 members)
- Duchenne Muscular Dystrophy (11 questions, 4 members)