SMA Europe Sets Out its Concerns about the AVXS-101 Zolgensma Global Compassionate Use Programme Announced in December 2019

06 January 2020

As members of SMA Europe, we endorse this statement made by SMA Europe on 26th December. It outlines the many concerns there are about this programme.

We are hopeful that AveXis will take up the offer made by SMA Europe, supported by its Scientific Advisory Board, to work jointly on improving the design of the programme so that this new therapeutic option is made available in a clinically justified, safe and fair manner. 

Read the statement