England to Screen All Newborns for SMA After Jesy Nelson Campaign; Real-World Zolgensma Data Show Motor Gains

All newborn babies in England will be screened for spinal muscular atrophy (SMA), a landmark change following former Little Mix singer Jesy Nelson's public campaign after her twin daughters were diagnosed with the rare genetic disorder. Nelson announced the policy change this week, saying it would help ensure no baby is overlooked and give future families the opportunity to access life-changing treatment as early as possible.

The screening program uses the routine newborn heel-prick blood test to identify babies with SMA before symptoms develop, a critical window because available treatments cannot reverse nerve damage that has already occurred. Nelson's daughters, Ocean Jade and Story Monroe, were diagnosed with SMA type 1, the most severe and common form of the disease. In January, Nelson revealed the twins had been diagnosed with the condition, which meant they would "probably never walk." She gave birth prematurely at 31 weeks in May 2025, and the girls had not been showing as much movement in their legs as they should have been and were struggling to feed properly.

Days after the announcement, Nelson's petition calling for SMA to be added to post-birth baby checks for serious health conditions passed 100,000 signatures, meaning MPs will now have to debate it in the House of Commons. In a video shared on Friday, Nelson was seen breaking down in tears saying: "You have no idea how much this means to me and the SMA community." Last month she also met the Health Secretary to speak about the life-changing impact early detection could have had on her twins. At present, screening for SMA is only carried out on those who have a sibling with the condition. SMA UK also wants it added to the newborn blood screening test, which is offered to every baby at five days old.

SMA is a progressive muscle-wasting disease caused by mutations in the survival motor neuron 1 (SMN1) gene, which encodes a protein essential for motor neuron survival. Without sufficient protein, motor neurons gradually die, causing progressive muscle weakness and wasting. The NHS says it can affect sitting up, crawling and walking, as well as problems with breathing or swallowing. SMA occurs in an estimated one in 10,000 births, and type 1 accounts for around 60% of all cases. There is still no cure for SMA, but medications and therapies can help manage the condition.

New real-world data add to evidence for Zolgensma (onasemnogene abeparvovec-xioi), a one-time gene therapy that delivers a healthy copy of the SMN1 gene to cells and is approved in the U.S. and Japan for children younger than 2. A study published in the Annals of Clinical and Translational Neurology, funded by Novartis Gene Therapies, analyzed real-world use and outcomes in 80 Japanese children with SMA, most with SMA type 1 (78.3%). The vast majority (93%) were alive and did not need permanent breathing support three years after receiving Zolgensma. Among 39 children with two or more motor milestone assessments, 64.1% achieved new milestones and 15.4% maintained their milestones. Milestones included sitting without support, standing alone, and walking alone. Those diagnosed through newborn screening achieved their first milestone four to 26 months earlier than children diagnosed clinically. The safety profile was consistent with earlier reports; all children had at least one adverse event, with the most common including fever, elevation of liver enzymes, decreased platelet counts, and vomiting. About two-thirds (68.8%) experienced serious adverse events, and one child died due to respiratory failure, considered unrelated to Zolgensma.

SMA has become one of the clearest examples of how newborn genetic screening is reshaping the treatment of rare diseases. For Nelson, the policy change would not change her daughters' diagnosis, but it could transform the lives of future children born with SMA.

In a separate development, a five-year-old girl from Snodland in Kent is taking on a 6.8km (4.2-mile) scooter challenge around Leybourne Lakes Country Park to raise money for her friend Sid, who has SMA type 1 and is a wheelchair user. Rosie has so far raised more than £3,400, surpassing her target of £2,000. Sid was treated with the £1.8m single dose of gene therapy Zolgensma, according to the charity Tree of Hope. His family needs a through-floor lift and an extension including an accessible bedroom and bathroom, work that would cost £138,000. Sid's mother said they were "grateful" for every donation and especially thankful to Rosie's "incredibly thoughtful" support.