SMA therapies available on the NHS

Children with the muscle-wasting condition spinal muscular atrophy (SMA) are to be offered therapy on the NHS which could help them live years longer and attend primary school. Two treatments for SMA – nusinersen and risdiplam – are to be routinely available on the NHS in England following an access scheme to collect more evidence, giving hundreds of families long term-certainty. NHS England has announced it had secured a commercial agreement to ensure lifelong treatment for anyone eligible, as data from the SMA REACH UK study found the therapies have now helped 73 children with the severe form of SMA (Type 1) survive to aged 5 or older – more than half of those treated.