A GENETIC illness that condemns children to a life of suffering and a premature death could be treated within five years with the support of Times readers. Spinal muscular atrophy (SMA), once among the least-understood diseases in medical textbooks, has recently emerged as one of the genetic conditions for which a therapy is almost within reach. Six thousand British children suffer from this devastating disease. The prospect of a cure or, at least, a treatment that can stop the disorder from getting worse has been transformed by a string of discoveries since 1995, many of them by British researchers. The pace of advance, however, will be heavily dependent on the level of funding that their work can attract. Experts agree that annual funding of just £10 million to £15 million worldwide would greatly accelerate the identification of potential treatments that could go into clinical trials within a year, but support, which relies largely on charities and government agencies, currently falls well short of this. As the first treatments are likely to work best when given to patients whose nervous systems have yet to be attacked by SMA, and one in every 6,000 children is born with the disease, every year of delay will have a heavy human cost.
