An exon-skipping medicine for certain types of Duchenne muscular dystrophy
Golodirsen
A specialist infusion used in certain types of Duchenne muscular dystrophy, where it helps the body make a more useful form of a missing muscle protein.
What is Golodirsen?
Golodirsen is a specialist medicine used to treat Duchenne muscular dystrophy, an inherited condition that causes progressive muscle weakness, in people whose specific genetic change can be helped by an approach called exon 53 skipping. It is given as a regular infusion into a vein. It works by helping the body produce a more useful form of dystrophin, the muscle protein that is missing in this condition. Important safety points include monitoring the kidneys and watching for allergic-type or hypersensitivity reactions. It is used only in people whose genetic test shows it is suitable.
Education and reference only. This is a plain-language guide to Golodirsen — it deliberately contains no doses. Doses depend on the person, the brand and the reason for treatment, and belong with your prescriber. Always check the BNF, the product labelling (SmPC) and follow medical advice.
What it is
Golodirsen is a specialist treatment for Duchenne muscular dystrophy, an inherited condition, mainly affecting boys, in which the body cannot make a working version of a muscle protein called dystrophin, leading to progressive muscle weakness. It is suitable only for people whose particular genetic change can be addressed by skipping a section of the gene known as exon 53, which a genetic test confirms. It is given as a regular infusion into a vein, under the care of a specialist neuromuscular team, as part of a wider plan of care.
How it works
In Duchenne muscular dystrophy, a fault in the dystrophin gene stops the body making the dystrophin protein that muscles need to stay strong. Golodirsen works by helping the cell's machinery skip over a specific faulty section of the gene (exon 53) when reading it, so that a shorter but more useful form of dystrophin can be produced. The aim is to provide some working protein and help slow the loss of muscle strength. It is given regularly because the effect depends on continued treatment, and it is only useful in people whose genetic change suits this skipping approach.
Company & origin
Originated / developed by: Specialist manufacturer.
A specialist medicine used to treat Duchenne muscular dystrophy in people whose genetic change can be helped by skipping a particular part of the gene (exon 53).
Practical use
How to take Golodirsen
General, dose-free guidance — always follow your prescriber's and the leaflet's specific instructions.
- It is given as a regular infusion into a vein at a specialist centre, on the schedule the neuromuscular team sets.
- It is used only if a genetic test shows your particular change can be helped by skipping exon 53.
- Attend appointments for regular kidney checks through blood and urine tests.
- Report any rash, fever, breathing changes or feeling unwell during or after the infusion straight away.
- Continue the rest of your care plan, such as steroids, physiotherapy and heart and breathing checks, as advised.
Weighing it up
Advantages & disadvantages of Golodirsen
Advantages
- Targets the underlying genetic cause in suitable people, helping the body make a more useful form of dystrophin.
- Offers a treatment option aimed at slowing muscle weakness in Duchenne muscular dystrophy.
- Used as part of a comprehensive specialist care plan.
Disadvantages
- Only suits people with a specific genetic change that can be helped by skipping exon 53.
- Given as a regular infusion into a vein, needing visits to a specialist centre.
- Needs kidney monitoring and carries a risk of allergic-type or hypersensitivity reactions.
Practical use
Good to know
A key point with golodirsen is that it only suits people with a specific genetic change that can be helped by skipping exon 53, so a genetic test is needed to confirm it is the right treatment. It is given as a regular infusion into a vein, which means visits to the specialist centre. The two main safety areas the team watches are the kidneys, with regular urine and blood tests, and the possibility of allergic-type or hypersensitivity reactions during or after the infusion, such as rash, fever or breathing changes, which are managed promptly if they occur. Golodirsen is one part of overall care, which also includes steroids, physiotherapy and support for the heart and breathing. It aims to slow the condition rather than cure it, and it is used under close specialist supervision.
Who should not take it / use with caution
- People who have had a serious allergic reaction to golodirsen should not use it.
- It is not suitable for people whose genetic change cannot be helped by skipping exon 53.
- It should only be used under a specialist neuromuscular service with the necessary monitoring.
Monitoring
- Regular blood and urine tests to check kidney function.
- Watching for allergic-type or hypersensitivity reactions during and after infusions.
- Reviewing muscle strength and overall progress as part of wider specialist care.
Side effects
- Headache, fever or cold-like symptoms.
- Reactions around the time of the infusion, such as rash, flushing or feeling unwell.
- Less commonly, signs the kidneys need watching, which is why regular kidney tests are done.
Key interactions
- There are few well-established routine medicine interactions, but tell your team about all your medicines.
- Other medicines that can affect the kidneys are reviewed, as kidney monitoring is important.
- It is used alongside, not instead of, the rest of the Duchenne muscular dystrophy care plan.
Available as: A solution for infusion into a vein, given at a specialist centre.
Answers
Golodirsen: frequently asked questions
What is golodirsen used for?
It is used to treat Duchenne muscular dystrophy in people whose specific genetic change can be helped by an approach called exon 53 skipping, confirmed by a genetic test.
How does it work?
It helps the cell skip over a faulty section of the dystrophin gene so that a shorter but more useful form of the muscle protein dystrophin can be made.
Is it suitable for everyone with Duchenne muscular dystrophy?
No. It only suits people whose particular genetic change can be helped by skipping exon 53, so a genetic test is needed first.
Why are kidney tests needed?
The kidneys are monitored with regular blood and urine tests during treatment, as this is an important safety check with golodirsen.
Does it cure the condition?
No. It aims to help slow the loss of muscle strength rather than cure the condition, and it is used as part of a wider specialist care plan.
Authoritative sources
- BNF
- NICE CKS
Building a medicines information resource?
We create evidence-led, dose-free drug and formulary references for teams.