NHS England to fund first ever treatment for children with rare muscle-wasting condition

NHS England and NHS Improvement will provide a promising new treatment which can prolong the lives of children with the rare genetic condition, spinal muscular atrophy (SMA). Nusinersen, also called Spinraza will be offered to those whose clinicians think they would benefit. While not a cure, trials have shown that nusinersen can slow the effects of SMA in some cases, allowing babies and toddlers to develop stronger muscles and survive for longer without breathing support.

Simon Stevens, NHS England’s Chief Executive, said: “This promising treatment has the potential to be life changing for children and their families”.