SynaptixBio is aiming to develop the world’s first treatment in the coming years. Launched last year, the company is working on a new therapy it hopes will “revolutionise” how TUBB4a leukodystrophy is treated.Antisense Oligonucleotides (ASOs) therapy, which has previously been used to treat other conditions, is also hoped to dramatically improve the quality of - and extend - the lives of leukodystrophy patients.SynaptixBio co-founder and CEO Dr Dan Willams said the treatment had the potential to “modify the disease, increase survival rates and significantly improve motor skills development.“The new approach provides the potential to stabilise, improve quality of life and extend life expectancy in children suffering from the condition,” he added.“Successful prevention of leukodystrophy progression would be a revolutionary life-saving and life-enriching treatment for children around the world.”Research has already begun, with the firm aiming to launch clinical trials in 2024.“This project will change people’s lives.
The research and development of a clinically-proven treatment for TUBB4a would be a real game-changer for patients and their families,” Dr Williams said“There is a real chance to improve the lives of leukodystrophy sufferers.
We want to ensure that dream becomes a reality.” For more information visit www.synaptixbio.comTo stay up to date with all the latest news, make sure you sign up for one of our free newsletters here..
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