Help for today, hope for tomorrow

The Jennifer Trust for Spinal Muscular Atrophy is the only national charity in the UK dedicated both to supporting people affected by SMA, and investing in essential research into causes, treatments and eventually a cure for the condition.

Important Notice

We're very sorry, but the Beach Day at Worthington on the 26th August 2010 has been cancelled due to bad weather.

Exciting Research News

The research team at Sheffield, led by Professor Mimoun Azzouz, tested whether a novel gene replacement therapy through a single injection was the most efficient way to treat the disease in a group of mice. They found that the injection, which expressed the SMN gene, successfully restored the SMN protein to normal levels and alleviated symptoms in the SMA model.

The new technique has the potential to develop a simple injection, without any requirement for risky and costly surgical interventions, and has achieved the highest therapeutic effects reported in the field to date. This has significant implications for the future treatment of SMA.

Read more about this gene therapy research breakthrough.

What is Spinal Muscular Atrophy?

Spinal muscular atrophy (SMA) is a condition that affects the nerves in an area of the spinal cord called the anterior horn. These nerve cells become damaged, breaking the link between the brain and the muscles. As a result, the muscles can't be used and become wasted or atrophied.