Families affected by spinal muscular atrophy are welcoming expanded NHS access to two treatments that specialists say are transforming outcomes for children diagnosed with the rare genetic condition.
The National Institute for Health and Care Excellence has approved the use of nusinersen and risdiplam for routine NHS treatment across England. Medical experts say the therapies can improve survival, slow the progression of the disease, and help patients maintain greater independence.
Spinal muscular atrophy, often referred to as SMA, is a rare inherited condition that causes muscle weakness and can affect breathing, movement, and swallowing. Around 70 babies are born with the condition in the UK each year, while more than 1,000 people are currently living with SMA in England.
One family from Ramsgate in Kent described the decision as life changing after their son Ezra responded positively to early treatment. Ezra began receiving nusinersen injections through an NHS early access programme when he was just five months old.
His mother Portia said the treatment had fundamentally changed the direction of his life. She explained that doctors initially warned the family their son might not survive beyond the age of two, making milestones such as attending mainstream school feel impossible at the time.
NHS England said dozens of children with SMA have already benefited from access to the treatments through specialist programmes. Health officials believe wider approval will now allow more families to receive support earlier following diagnosis.
Medical organisations and charities supporting people with SMA have described the approval as a major step forward for treatment options in the UK. Health experts say earlier diagnosis and faster access to medication can have a significant impact on long term outcomes.
Campaigners are also continuing efforts to increase awareness of SMA and improve newborn screening programmes. Public discussion around the condition has increased in recent months following awareness campaigns involving singer Jesy Nelson, who previously spoke publicly about SMA affecting her daughters.
Healthcare leaders say the wider rollout of the treatments represents an important development in rare disease care and could continue improving quality of life for many patients and families across England.