Published Date
Clare Lucas
Developing treatments

We are aware that complex and sensitive information regarding potential but very limited global access to the gene therapy treatment Zolgensma for SMA has been recently shared by AveXis online with the medical and advocacy group community of many European countries and is being discussed within the SMA Community.

We are conscious that no official public information is as yet being provided and that UK clinicians have yet to receive information from AveXis regarding how and if this process could be available to patients in the UK.

Clinicians and patient groups have expressed their concerns to the drug company AveXis requesting, as a matter of urgency,  public clarification of the position. We consider it vital that any information that is shared is as full and accurate as possible.

We understand the stress and anxiety created when there is uncertainty about potential new treatments – SMA UK has provided information for people who are considering seeking access to treatment that is not currently available to them, which you can read at:

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