There is new hope for patients and their families suffering from spinal muscular atrophy (SMA) reported AOL UK News. SMA is a physically debilitating and aggressive disease, and now a new treatment called Spinraza might have positive effects and be available sooner for these patients who’s lives are at major risk.
SMA affects nearly 1,300 individuals in the UK, and every year there are 40 newborns with the disease. Life expectancy for these children are only two years, but access to Spinraza would significantly increase their expectancy, as well as improve their quality of life. To learn more about this rare disease, click here.
The drug has shown positive responses to children, aiding in a decrease of mobility deterioration, as well as give them the opportunity to develop new skills. Muscle improvement has been shown in clinical trials, and some babies who were expected to face death at two-years-old are now crawling and walking.
Liz Lockley, a mother of a two-year-old with SMA Type Two, shared that the drug has allowed her son George to maintain the strength he has built up. While this is great news since it decreases the intensity of the disease and gives patients the opportunity to experience growth, the drug is not a cure.
Yet sometimes, it’s the small wins that count and make the biggest impact. Now little George has arm functionality, allowing him to be more independent. Spinraza has continued to provide children with success through the clinical trial so greatly that the researchers stopped the trial and continue to push it forward faster so that other children can access it sooner.
There is much more that needs to be done. Not every child who needs the drug treatment has access to it, but the mission continues to be in progress and push forward to get to their goal of widespread reach. Muscle atrophy is scary for anyone. Seeing a loved one slowly lose their muscle function and ability to be independent is devastating. Hopefully, Spinraza continues to flourish and reach the masses as soon as possible.