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| 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 |
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.