Hundreds more children with spinal muscular atrophy (SMA) can now access life-changing NHS treatments after the National Institute for Health…
Browsing: Gene Therapy
England will screen 750,000 newborns for spinal muscular atrophy as part of a landmark study to determine whether the genetic…
A six-year-old girl from Hertfordshire has had her sight restored by a pioneering gene therapy on the NHS, a treatment…
Two young brothers from west London are in a race against time to find the stem cell donors who could…
The national rollout of screening for the rare genetic condition spinal muscular atrophy (SMA) in newborns is to begin earlier…
A toddler from Kent faces a desperate race against time for a life-saving treatment, with a crucial window of just…
A two-year-old girl is in a race against time, facing the imminent loss of her ability to walk, talk, and…