Page last checked: 18th December 2024

Reference  –NICE’s website

Your baby must:

Have had a confirmed diagnosis of 5q autosomal recessive SMA – an SMN1 gene deletion test and a clinical diagnosis of SMA Type 1.

If your baby:

• is 6 months or younger, they may be referred directly by a Regional Neuromuscular Centre (RNMC) for access to treatment at an Infusion Centre
• is aged 7 to 12 months, their treatment has to be first agreed by the National Multidisciplinary Team (NMDT).

Your baby must not be on permanent ventilation for more than 16 hours per day or have a tracheostomy.

OR

Be pre-symptomatic with a confirmed diagnosis of 5q autosomal recessive SMA – an SMN1 gene deletion test – and up to 3 copies of the SMN2 gene. They must be aged 12 months and under.

Children with SMA Type 1 who are over 12 months old within the scope of the drug’s European Medicines Agency (EMA) marketing authorisation.

Additionally, in 2021, NHS England approved treatment for children who have SMA Type 1 who are over 12 months old and within the scope of the drug’s European Medicines Agency (EMA) marketing authorisation. Most of these children have now been assessed for treatment by the NMDT. See: What is a safe weight or maximum age for treatment with Zolgensma – Dropdown tab 9 >.

Age Definitions:

Aged six months and younger refers to calendar months and includes children below 7 months of age (e.g. if a child is born on the 1st January 2025, they will be 7 months old on the 1st August).

Aged 7 – 12 months refers to calendar months and includes children who are 7 months old up to when they turn 13 months (e.g. if a child is born on the 1st January 2025, they are 7 months old on 1st August and turn 13 months old on the 1st February 2026).