Dr Heidi Fuller and Dr Sharon Owen presented their research into identifying treatment strategies for children with spinal muscular atrophy (SMA) at the launch event of No Time to Lose, a campaign by children’s medical research charity Sparks.

Dr Fuller and Dr Owen, from Keele’s Institute of Science and Technology in Medicine, hosted a stand about their Sparks and Great Ormond Street Hospital Charity (GOSH) funded research project which looks to identify treatment strategies that will likely be effective for children with Type II and III SMA, a form of motor neuron disease that leads to muscle wasting. This could lead to new and more effective treatments for this devastating condition and pave the way for a cure.

Through the No Time to Lose campaign, which was introduced at the launch event by Sparks children’s champion, Gabby Logan, Sparks aims to raise £10 million over the next four years to help find the treatments children with a rare condition urgently need.

Dr Fuller said: “It was such a privilege to attend the launch event and to meet families affected by rare diseases. As scientists, it is so important we remain focused on why we do what we do.”

“We are grateful for the opportunity to talk to Sparks supporters about our research project and were blown away by their enthusiasm and engagement!

“During our Sparks and GOSH funded project we aim to understand differences between the different types of SMA. This work will help to inform the design of clinical trials for SMA, and will also help to understand whether the treatment strategies that are being developed for babies with severe SMA will likely be effective for children with the other types of SMA.”

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