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Clinician advice – SMA Gene Therapy

SMA Gene Therapy

Welcome to the referral page for Sheffield Children’s NHS Foundation Trust SMA Gene Therapy Service.

Zolgensma® is currently available for:

  • Children under 13 months of age diagnosed with SMA type 1 or who are identified as pre-symptomatic of having SMA (and have three or less copies of the SMN2 gene) who are not on any other SMA drug treatment 
  • Children with SMA type 1 or pre-symptomatic of having SMA under 13 months of age who are on other SMA drug treatment.

Some of the patients will need to be discussed in a national meeting and as such not all children will meet the criteria and may not be able to access this treatment.

Prior to referring a child for Zolgensma®, we need copies of the following information:

  • Renal function
  • Liver function
  • Full blood count
  • Troponin I result
  • Anti_AAV9 antibody results
  • Copy of an ECG (if possible, but this should not delay referral)
  • Echocardiogram result (if possible, but this should not delay referral)
  • Vaccine summary
  • Medical summary
  • Physiotherapy assessment using SMAREACH standards (if possible)
  • A medical summary

We will also need you to complete a referral form, which can be found online via the referral portal.

Please also call the following number to talk to our administration assistant if you have any queries on 07468 351 090 between working hours (9am to 5pm) Monday to Friday. You can contact the Neuromuscular Consultants for clinical enquiries via switchboard on 0114 271 7000 – ask to talk to Dr Ong or Dr Atherton.

Children in Group A will be infused without discussion in the national MDT meeting. If there is no capacity in a local centre, we may need to find another centre who can organise treatment. Other patients will be discussed in the national MDT meeting, and you will be informed of the results of that discussion and priority/likely timing of infusion.

If your patient is eligible, we will be in touch will them to arrange a video appointment to discuss the treatment and what is required.

A week before the infusion date, you will need to check the patient is well, free from infection and to get an up-to-date weight of the child. We require this up-to-date weight information (must be naked weight) to order the correct drug dose. Please ensure that this information is emailed through to our Neuromuscular administrator at

We aim to arrange treatment one to two weeks later on our High Dependency Unit. The family will be allowed to go home after four days if they live within 30 minutes’ drive of Sheffield Children’s Hospital. If they live further away, we will provide alternative accommodation. However, in exceptional situations, we may be able to agree shared care with you so the family can go home. Please do talk to the consultants about this possibility. The children will then have regular appointments over the first two weeks and, if everything is OK, will be allowed home at this point.

We will arrange regular follow up reviews of the child post treatment, with the minimum being three months.

As we gain more experience of the treatment, our pathway may change. Our current pathway for treatment is available on the SMA Gene Therapy Service homepage.

Return to SMA Gene Therapy Service homepage
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