Queen Square Centre for Neuromuscular Diseases


Outcome measure in SMA type II and III

Sponsor UCL Institute of Child Health
Funder SMA Europe
CI Professor Kate Bushby, Professor Francesco Muntoni
Sites Newcastle, London GOSH
Contact details

Newcastle: becky.davis@ncl.ac.uk

London: ich.neuromusculartrials@ucl.ac.uk

This prospective longitudinal natural history study will be performed in two groups of patients with SMA type II and III. 

The first group is ambulant patients and the second non-ambulant. Inclusion criteria and methods will be different for the two groups.

We have considerable retrospective data on SMA but little research data and none using the range of outcome measures proposed. The aim is to be ready for future multi-centre trials on SMA type II and III

Primary objective

To establish a clinical network which will enable common outcome measures on SMA type II and III. The network should involve most of the leading neuromuscular centres in Europe.

Secondary objectives

To ensure:

  • functional scales used are suitable and clinically relevant for future trials,
  • we understand how the different measures relate to one another and how they may change over a 12 month period.