NHS professionals speaking to paediatric nurse

New NHS treatments boosting outcomes for babies with rare genetic condition

Treatment advancements are helping improve health outcomes for babies with spinal muscular atrophy (SMA), NHS England has announced.

SMA is a rare genetic condition that leads to muscle weakness and progressive loss of movement and paralysis – approximately 70 children are born with the disease in the UK every year.

Previous studies have indicated that, before the availability of treatments, fewer that one in 10 (8%) of those born with the most common form of the disease – SMA type 1 – survived to the age of 20 months without permanent ventilatory support.

However, thanks to new NHS treatments, almost three-quarters (73%) of children born with SMA type 1 are now older than two-years-old.

This data comes from the national SMA research and clinical hub database, which also suggests a significant drop in mortality – only 11 deaths were reported in the UK between March 2018 and March 2023, compared to around 25 a year in England between 2008 and 2017.

                                                                      Video credit: Canva

The data highlights the impact of the drug nurinersen, which was the first treatment to target the underlying cause of SMA, alongside the gene therapy Zolgensma® and oral treatment risdiplam which mean the NHS has a treatment for SMA type 1, 2 and 3.

National medical director of specialised services at NHS England, Professor James Palmer, added: “A trio of new treatments are improving outcomes for NHS patients by slowing the progression of SMA, enabling a longer and better quality of life with family and friends.

“It’s brilliant to see the life-changing impact these drugs are already having and there is every reason to believe we will see an even greater impact over time, especially when babies receive treatment at the earliest opportunity.”

Image credit: iStock

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NHE May/June 2024

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