A vital drug could be banned for use in the NHS and that would be devastating for about 1,000 people with the rare disease of spinal muscular atrophy (SMA).
The National Institute for Health and Care Excellence (NICE), which provides guidance and advice to improve health and social care, has issued draft guidance on the Spinraza drug, which says it should not be used on the NHS for cost and other reasons.
The manufacturer of Spinraza has announced that special compassionate access will be closed to new patients from November 1.
Without treatment, babies are unlikely to see their second birthday, but Spinraza means some have gone on to crawl and even walk.
As the chairman of the All Party Group on Muscular Dystrophy, I know that this is extremely upsetting news for the families affected and I am working urgently with the Muscular Dystrophy UK charity to push for this vital treatment to be allowed in NHS treatment.
NICE’s final decision is expected in November, or early next year.
And my priority when parliament returns next week is to help bring together all those concerned to make the case for Spinraza.
I will chair an emergency meeting with MPs, NHS England, the manufacturer and NICE and enable parents and carers to share their personal stories of life with SMA with MPs. I will also urge the Prime Minister to meet me and others to find suitable solutions to afford these children the best chance of life.