This video from Nemours Children’s Hospital shows the effects of the drug nusinersen (Spinraza) on a young spinal muscular atrophy (SMA) patient.
Three-year-old Asher Camp from Florida was diagnosed with type 1 SMA when he was six months old, and was subsequently enrolled in the clinical trial for nusinersen. As you can see, the transformation since taking the drug is remarkable and he is now doing things his doctors and parents never dreamt he would be able to.
The team at Nemours Children’s Hospital are continuing to work with the drug to establish if it could prevent the progression of SMA if given at an earlier age. Nusinersen (Spinzara) was approved by the FDA for use for children and adults with SMA on December 23, 2016, and gives hope to thousands of families across the country.
SMA News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.