First UK baby successfully receives lifes-changing treatment for SMA


The first infant to be treated with a ground-breaking gene therapy for spinal muscular atrophy (SMA) is currently recovering at Sheffield Hospital.

Two month old Markuss was diagnosed with SMA type 1 after his health visitor picked up on red flags for the condition soon after his birth.

The early recognition and subsequent diagnosis have been life-saving for Markuss, who was then eligible for the brand new gene treatment Zolgensma. The one-off treatment, delivered intravenously, can potentially enable an infant with SMA type 1 to reach all of their normal milestones - something unprecedented until just this year.

65 babies are born annually with SMA, 60% of whom will have type 1 like Markuss - the most severe form, affecting a baby before they reach 6 months of age.

Markuss is currently recovering well, and this treatment, along with education and support to help professionals detect, diagnose and treat babies like Markuss in the future, will hopefully make him the first of many whose lives are changed for good.

More information

  • Do you know the red flags for SMA type 1? Watch our short video - these 3 minutes could save a life.

  • What is your own role in supporting babies and children with SMA? Find out in our new paediatric pathway.

Raising awareness, improving outcomes

Neuromuscular Academy is the first bespoke course for healthcare professionals to receive expert training in neuromuscular conditions like spinal muscular atrophy.