NHS England | Zolgensma Gene Therapy Referral Pathway |

BEFORE COMPLETING A REFERRAL  PLEASE READ THE FOLLOWING

This referral form should be used to make referrals for children:

  • with a negative antibody titre for AAV9 (less than 1:50) or test currently underway

  • without tracheostomy or permanent ventilation for more than 16 hours per day

  • under 21kg body weight (note in the first year of access invitations will be by weight band)

Additional criteria are per the NICE guideline or NHS England Managed Access Agreement as follows:

  • SMA Type 1 with genetic test result confirming 5q SMA with a bi-allelic mutation in the SMN1 gene and under 13 months of age or;

  • pre-symptomatic and up to 3 copies of the SMN2 

To expedite treatment planning referrals are accepted for children whose AAV9 result is pending.

Only RNMCs may refer patients for infusion. Infusion centres and the National MDT cannot receive referrals from other providers. If you are not an RNMC please refer the patient to the patient’s nearest RNMC.

 

Patients will require a local workup by their RNMC. The referral form will ask for details of this workup (eg physio assessment, genetic and blood test results) to enable prioritisation and eligibility assessment by the National MDT.

 

RNMCs are able to start referral forms and save them in draft so extra details can be added at a later time. Note that until all mandatory fields are completed and the form submitted, the referral WILL NOT be made available to infusion centres/the National MDT.

 

RNMCs are advised to use the correct referral forms as follows:

Treatment naïve patients aged under 7 months (ie. 6 months and 31 days and under)

 

Pre-symptomatic patients with up to 3 copies of the SMN2 gene who are treatment-naive

 

referred directly to the preferred infusion centre for treatment.

It is highly advisable to ALSO make a phone call to the infusion centre for case discussion – this will aid planning for transport.

 

In choosing a preferred infusion centre, give consideration to distance for the family, and family choice.

All patients already on treatment (regardless of age)

 

Treatment naïve patients aged 7 months and over

 

will be referred to the National MDT.

The National MDT will assess eligibility and prioritise accordingly. The referrer will be informed of the National MDT decision, and (if appropriate) the allocated infusion centre. Wherever possible the preferred infusion centre will be allocated (specified on the form) – please note this is subject to available capacity.

To make a referral you will require your patient's 10-digit NHS number and a Trust or nhs.net email address. referapatient® works best when you use a modern browser such as Google Chrome or Mozilla Firefox

 

To speak with the infusion centre directly please contact:

​Royal Manchester Children’s Hospital, Manchester University NHS Foundation Trust

0161 701 0679 (direct), mft.nmteam@nhs.net or 0161 276 1234 (switchboard)
 

Sheffield Children’s NHS Foundation Trust

07468351090 (Mon-Fri 9-5 ) or 0114 271 7000 (switchboard for on call consultant)
 

Evelina Children’s Hospital, Guy’s & St Thomas’ NHS Foundation Trust

gst-tr.evelinaonasemnogenereferrals@nhs.net
 

University Hospitals Bristol NHS Foundation Trust

jane.berry3@Uhbw.nhs.uk 

AFTER COMPLETING A REFERRAL 

 

You will receive acknowledge of referral by email and can find your referral in the 'Referrals I have Made' section after you log in.

 

​Should referrers become aware of deterioration/infection in the patient before they attend the infusion centre, this should be notified to the infusion centre as soon as possible so delivery of the dose can be amended if required.

 

Following infusion, blood tests will be needed on a regular basis. Results of these tests MUST be communicated to the infusion centre as part of the safety monitoring. By referring a patient the referrer agrees to communicate blood test results to the infusion centre.