You may be familiar with the work of the Oxford centre – it was founded in 2019 as a partnership between MDUK and the University of Oxford, with the goal of driving forward the development of novel experimental therapies. In his role, Professor Servias is applying his experience and expertise to the search for more and better treatments for muscle-wasting conditions:
If you had told me back in 2008, that by 2021 we would have the ability to effectively treat an entire disease by screening for it from birth, I would not have believed you. And yet data shows that this may soon be possible. In the case of spinal muscular atrophy (SMA), if we can identify the condition in newborns, we can treat it immediately using gene therapy.Advances like this excite me tremendously. We still have more to learn about the long-term efficacy of this approach, but the data so far is extremely promising. It is my profound hope that this combination of screening and treatment will prove truly effective, and that it will be made available in the UK.
MDUK is a leading member of the Newborn Screening for SMA Alliance, based in Oxford, and we are working hard to convince the National Screening Committee to permit screening for SMA at birth.
Gene therapy is a powerful tool, and we are only just beginning to understand the full range of its uses.
I firmly believe we could be heading towards a world in which we can diagnose and treat more neuromuscular conditions far more effectively, from birth, in much the same way as we may soon be able to do with SMA.
Incredible progress is being made, but we need your help so that we can continue to fund excellent research to make these latest breakthroughs available in the UK to those who need them. Please donate today and be part of something truly transformational. Thank you.