A spinal injection treatment for spinal muscular atrophy
Nusinersen
A treatment for spinal muscular atrophy, given into the fluid around the spinal cord by lumbar puncture.
What is Nusinersen?
Nusinersen is a specialist medicine used to treat spinal muscular atrophy, a rare inherited condition that weakens the muscles by affecting the nerves that control them. It is a type of antisense oligonucleotide that helps the body make more of a protein the nerves need. It is given into the fluid around the spinal cord by lumbar puncture, with starting doses closer together and then maintenance doses spaced out. Because of how it is given and its effects, the team checks blood clotting, platelets and kidney function during treatment.
Education and reference only. This is a plain-language guide to Nusinersen — 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
Nusinersen is a medicine used to treat spinal muscular atrophy, an inherited condition in which the nerve cells that control muscles are lost, causing progressive muscle weakness. It is a type of antisense oligonucleotide, a medicine that changes how the body reads a specific genetic message. It is given into the fluid that surrounds the spinal cord, through a procedure called a lumbar puncture (a spinal injection into the lower back), so it can reach the nerves directly. It is prescribed and given by a specialist team experienced in this condition and the procedure.
How it works
In spinal muscular atrophy, there is too little of a protein that the motor nerve cells need to survive, partly because a back-up gene is not read properly. Nusinersen works by helping the body read that back-up gene correctly, so it produces more of the needed protein. With more of this protein, the motor nerve cells are better supported, which helps maintain or improve muscle strength and function. Because the medicine needs to reach the nerves of the spinal cord, it is given into the surrounding fluid by lumbar puncture, with starting doses given closer together and then maintenance doses spaced out over time.
Company & origin
Originated / developed by: Specialist manufacturer.
A specialist treatment used in the UK for spinal muscular atrophy, given into the fluid around the spinal cord.
Practical use
How to take Nusinersen
General, dose-free guidance — always follow your prescriber's and the leaflet's specific instructions.
- Attend the specialist centre for each dose, as it is given into the fluid around the spinal cord by lumbar puncture.
- Expect starting doses closer together and then maintenance doses spaced out over time.
- Have the blood tests the team arranges, which check blood clotting, platelets and kidney function.
- Report unusual bruising or bleeding, as the medicine can affect clotting and platelets.
- Keep attending appointments, as this is a long-term treatment that needs ongoing review.
Weighing it up
Advantages & disadvantages of Nusinersen
Advantages
- Treats spinal muscular atrophy by helping the body make more of the protein the nerves need.
- Delivered directly to where it is needed, into the fluid around the spinal cord.
- Can help maintain or improve muscle strength and function over time.
Disadvantages
- Given by lumbar puncture, a spinal injection that some people find uncomfortable.
- Can affect blood clotting, platelets and the kidneys, so blood tests are needed.
- Requires repeated procedures at a specialist centre and is a long-term treatment.
Practical use
Good to know
The most distinctive thing about nusinersen is how it is given: into the fluid around the spinal cord by lumbar puncture, a spinal injection into the lower back, done by a specialist team. Treatment starts with several doses closer together and then settles into maintenance doses spaced out over time. Because of how it works and how it is given, the team carries out specific blood tests around the procedure: checks on blood clotting and the platelets (the cells that help blood clot), since changes can occur, and checks on kidney function, as the medicine can affect the kidneys. Some children and adults may find the lumbar puncture itself uncomfortable, and the team takes steps to make it as comfortable and safe as possible. It is a long-term treatment that supports nerve and muscle function rather than curing the condition, and the team reviews progress over time.
Who should not take it / use with caution
- People who have had a serious allergic reaction to nusinersen should not have it.
- It is used with care in people with bleeding or clotting problems, which is why these are checked.
- It is used with care, and under specialist guidance, in pregnancy.
- It should only be given by a specialist team able to perform the procedure and the necessary monitoring.
Monitoring
- Blood tests around the procedure to check blood clotting and the platelet count.
- Checking kidney function, including a urine test, during treatment.
- Reviewing muscle strength and overall function over time to judge how well it is working.
Side effects
- Effects related to the lumbar puncture, such as headache or back pain after the procedure.
- Changes in blood clotting or platelets, which blood tests are designed to catch.
- Effects on the kidneys, checked with regular tests, and respiratory infections in some people.
Key interactions
- It is used with extra care alongside medicines that thin the blood or affect platelets, because of the clotting checks.
- Tell your team about everything taken, including over-the-counter products and supplements.
- There are few other well-established routine medicine interactions, but a full list is still important.
Available as: A solution given into the fluid around the spinal cord by lumbar puncture.
Answers
Nusinersen: frequently asked questions
What is nusinersen used for?
It is used to treat spinal muscular atrophy, helping the body make more of a protein that the nerves controlling the muscles need.
How is it given?
It is given into the fluid around the spinal cord by lumbar puncture, a spinal injection into the lower back, done by a specialist team.
How often is it given?
Treatment starts with several doses closer together and then settles into maintenance doses spaced out over time.
What blood tests are needed?
The team checks blood clotting and the platelets, as changes can occur, and checks kidney function, as the medicine can affect the kidneys.
Does it cure the condition?
No. It supports nerve and muscle function over the long term rather than curing the condition, so ongoing treatment and review are needed.
Authoritative sources
- BNF
- NICE CKS
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