Improving standards of care and facilitating clinical trials for spinal muscular atrophy

We are delighted to announce that we have partnered with The SMA Trust to support the ongoing development and expansion of SMA REACH UK. This will continue to improve standards of care for people living with spinal muscular atrophy (SMA) in the UK and will help to facilitate clinical trials.

The SMA REACH UK project was initiated to boost collaborative research and create the infrastructure, clinical networks and databases required in order to prepare the SMA field for clinical trials.

Since its conception in 2013, the project has combined the information held in the UK SMA Patient Registry and the SMArtNet database to form a single comprehensive database, which is regularly updated at patient clinic visits. This longitudinal data can be used for research purposes to improve clinical standards and to identify eligible individuals for clinical trials.

The project has also helped to establish a collaborative partnership between the Dubowitz Neuromuscular Centre, Newcastle University, the Italian SMA network and the Paediatric Neuromuscular Clinical Research Network (PNCRN) in the US. This has led to the revision of clinical assessment scales used to measure muscle function in people with SMA. These sorts of measurements are called outcome measures and are important for assessing the effect of new drugs in clinical trials, as well as for improving care standards.

SMA REACH UK received its initial £300K funding from The SMA Trust. Muscular Dystrophy UK has now joined forces with The SMA Trust to fund the second part of the project, which will cost £218,903 over two years.

The second part of the project aims to expand the SMA clinical and research network in the UK by engaging more centres, recruiting more patients and collecting more data.  It will also include Type 1 patients for the first time. It will continue to collect data based on the revised clinical assessment scales and evaluate their validity and reliability as outcome measures for SMA. It also aims to identify biomarkers that could be used to monitor the progression of the condition.

Dr Jenny Versnel, Director of Research and Business Innovation at Muscular Dystrophy UK, said:

The SMA REACH database is a valuable resource for clinicians, scientists, and industry wishing to carry out research or clinical trials. It is developing validated tools that can be used to assess the efficacy of interventions or treatments, which will help to pave the way for clinical trials. Muscular Dystrophy UK is delighted to be involved in a project that aims to improve the lives of people with SMA.

Joanna Mitchell, Chief Executive Officer at The SMA Trust, said:

We are delighted with the progress that has been made since we first supported SMA REACH and pleased that this important network can now expand further, both within the UK and through ever closer alliances with international networks. Standardised measurement and assessment through networks such as SMA REACH are vital amidst growing numbers of clinical trials and a real prospect of treatments for SMA in the foreseeable future.

Watch Professor Francesco Muntoni, who is leading the SMA Reach project at UCL Institute of Child Health:

Further information

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