Jessica May Purrington

Jessie May welcomes the news today that the National Institute for Health and Care Excellence (NICE) have approved a life-saving drug to treat babies with a rare fatal genetic disorder. The gene therapy, Zolgensma, offers hope to families of infants with a severe type of Spinal Muscular Atrophy (SMA).

Jessica May Purrington herself, the little girl who was the inspiration behind the founding of Jessie May, suffered from SMA Type 1, and died aged just 4 ½ months old.

The gene therapy itself has a list price of £1.79m, meaning that it could become the most expensive drug ever to be approved by NICE. However, NHS England are said to be negotiating an undisclosed discount that is said to be “fair to taxpayers”.

It is said that around 80 babies and young children across England who are born with Type 1 SMA could benefit from this treatment each year. It is hoped that if the drug is given to children in the first 6 months of their life, before they develop symptoms, that it might come as close to being a cure as possible by halting the progression of the disease.

CEO of Jessie May, Chris Roys, was full of delight at the news;

“This incredible news gives some much-needed hope to so many children and families. This drug may be able to help some of the children and families that our specialist Jessie May nurses support, both at present and in the future. Some may say that the treatment is very expensive, but is there really a price that you can put on a child’s life?”

At the moment there is very limited data about the impact of the Zolgensma therapy from clinical trials undertaken, and so any decision to give it to older children between 6-12 months in age will need to be discussed with your clinician.

To find out more about the Zolgensma drug, head over to