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Q and As About Nusinersen - Is It Appropriate For My Child?

SMA Support UK has been asked many questions by families and invited the SMA Trust and Muscular Dystrophy UK to add any additional ones to the list. SMA Support UK then liaised with clinicians who had been involved with the clinical trials and with Biogen for accurate technical answers to the questions that required their input.

These are the responses we have had as of 9th March 2017 and have added to as more information has become available:

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Q.1. Who has nusinersen actually been tested on (age / SMA Type / numbers)?

A. Answer

Q.2. Where can we find more detail of the actual nusinersen trial results?

A. The European Medicines Agency (EMA) Assessment Report received by NHS England week of 19th June gives a very full and detailed summary of all the clinical trials results to date. You can read this here. You can also read our Scientific Research Correspondent's summaries of key results here 

Q.3. How effective is nusinersen - what will it actually do for my child?

A. Answer

Q.4. How is nusinersen administered?

A. Answer

Q.5. Are there any known side effects to nusinersen?

A. Biogen state that no medicine is free from side effects and as very limited data on the safety profile of nusinersen are available in the public domain, they are unable to provide a response to this question. They ask people to consult their healthcare professional for further information on the side effects of nusinersen. Should the healthcare professional need any further information, he/she may contact the Biogen Medical Information Department (0800 008 7401).

Q.6. Is there any information available on the long term effects of nusinersen?

A. As the first in human study started in 2011 (NCT01494701), the long term effects are not yet known. Information about outcomes continues to be gathered.

Q.7. Is nusinersen a cure for SMA?

A. Answer

Q.8. Is it true that nusinersen is more effective the younger the person is when treatment is started?

A. Answer

Q.9. Will children who have a tracheostomy be able to access the treatment? 

A. Answer

Q.10. As it is to be administered by lumbar puncture will anyone who has had spinal surgery be able to access nusinersen? 

A. Answer

Q.11. I’ve heard that anyone with SMA may have low bone density and be more vulnerable to fractures. Can I get this checked and, if my child’s bone density is low, will this affect their chances of being treated by nusinersen? 

A. Answer

Q.12. What about those who take salbutamol? Does this affect their possible treatment with nusinersen?

A. Salbutamol is being administered more commonly to individuals with SMA. We understand that it would not prevent treatment with nusinersen. 

Q.13. If I / my child starts being treated with nusinersen, how long will I / my child need to stay on the treatment?

A. Answer

Q. 14. If my child became very unwell, would treatment with nusinersen be stopped completely? 

A. The lumbar puncture is a complex procedure which can only be performed if children are well. If a child is too unwell, the lumbar puncture will need to be rescheduled.

We understand that, if a child remains unwell or the treatment is not having any beneficial effects after a reasonable interval, the medical team and family would discuss whether or not to carry on with the treatment.

Q.15. How effective is nusinersen for adults with SMA? Has nusinersen been tested in adults?

A. There have been no clinical trials to date on adults with SMA so the impact is unknown.

Q.16 I have distal /another rarer form of SMA. Would nusinersen help me?

A. Answer

Q.17. What will be the options available for those not eligible for nusinersen?

A. Answer