NHS Confirms Long-Term Access to Life-Changing SMA Treatments

 The NHS has confirmed that two disease-modifying therapies for SMA—nusinersen and risdiplam—are now routinely available following years of managed access arrangements and growing clinical evidence showing improved outcomes for patients. The decision marks a major shift from temporary funding to permanent provision on the NHS. 

For families, including those in Greenwich supported through local GP surgeries and specialist hospital pathways such as those linked with Guy’s and St Thomas’ NHS Foundation Trust and Lewisham and Greenwich NHS Trust, the change removes long-standing uncertainty about whether treatment would continue beyond limited funding schemes. 

What the treatments involve 

Nusinersen is given as a regular injection into the spinal fluid in hospital, while risdiplam is taken daily at home as a liquid medicine. Both treatments work by increasing levels of a protein essential for muscle function, helping to slow or stabilise the progression of SMA.

Clinical data gathered over several years shows children treated early are now reaching developmental milestones that were previously unlikely, including improved breathing, movement and the ability to attend school. 

Why this matters locally 

SMA is a rare genetic condition that can cause severe and progressive muscle weakness. Without treatment, the most severe form is often life-limiting in early childhood. The new NHS approach reflects a shift towards earlier intervention, long-term continuity of care, and improved quality of life. 

For families in Greenwich, the change means treatment decisions are now part of standard NHS care pathways rather than time-limited access programmes, providing greater stability for long-term planning and support. 

Getting help and support 

Families concerned about symptoms or diagnosis of SMA should speak to their GP for referral to specialist neuromuscular services, typically provided through tertiary NHS centres in London. 

Further support and information is available from: 

• Muscular Dystrophy UK – 0800 652 6352 

• SMA UK (Spinal Muscular Atrophy UK) – 01789 267 520 

• NHS 111 for urgent medical advice (111.nhs.uk or 111 by phone).

   

Specialist care for SMA is usually coordinated through paediatric neurology and neuromuscular teams at London teaching hospitals, with ongoing support from community health services depending on individual needs.