What is SMA?

Spinal muscular atrophy (SMA) is a rare genetic condition that makes the muscles weaker and causes problems with movement. Type 1 is the most severe form and symptoms develop very early and cause death usually before the baby reaches two. Although the progression of the disease can be slowed with Spinraza (a drug offered by the NHS), the only cure that's available is gene therapy, which is sold under the name of 'Zolgensma' by Novartis.

The ultimate effect of Zolgensma will be to replace the mutated SMN1 gene with a fully functional one. SMA children's marrow and muscle cells will naturally start producing the essential protein SMN.

Further details can be found on:
NHS Website


Our Mission

For (SMA) type 1 childrens gene therapy (Zolgensma), we aim to reach £1.9m per child to give each of the children diagnosed with this rare disease a chance in life. Please donate as much as you can – even if it’s a £1 a week – and give these children the life they rightly deserve. Your help and contribution is not just a donation, but it is heroic as it will give a child, a whole family a second chance. All donations will be greatly appreciated and will always be remembered.

These children’s lives are dependent on your donations and we cannot succeed without you.

Please also share with anyone you feel would be willing to help.

Thank you!