Clinical trials in SMA

Promising new treatments for SMA are being developed. They need to be tested in clinical trials. The registry has been set up to speed the process of finding patients that fulfill the eligibility criteria for the trials.

When recruitment for an SMA trial starts in the UK, the UK SMA Registry will be in touch with those on the registry to let them know about it.

You can  also find the information about the current SMA related trials on the TREAT-NMD website:

The most comprehensive online listing of trials is at where companies are obliged to provide details of their trials.

A useful overview of the current trials can also be found on the SMA Support UK website here:

Update on the ENDEAR trial, Expanded Access Programme and the approval process for nusinersen (Spinraza)

10 May 2017

Updates relating to the Expanded Access Programme for Nusinersen in SMA Type 1 can be found on the UK SMA Support Uk website here:

21st April 2017

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the granting of a marketing authorisation of nusinersen, or Spinraza™ as it will now be called, for the treatment of "5q spinal muscular atrophy". The CHMP opinion forms the basis for a European Commission decision on whether to grant a licence for nusinersen. The official EMA's statement can be seen here.

21 December 2016

A joint statement has been released regarding the Expanded Access Program for Nusinersen in SMA Type 1 from London and Newcastle, in collaboration with the patient organisations. You can read this on the SMA Support UK website via this link: Update on nusinersen Expanded Access Programme in the UK

16 September 2016

The ENDEAR trial Principle Investigators in London and Newcastle have issued a statement on the ENDEAR trial and Expanded Access Program (EAP). You can download the statement  here.