SMA Clinical Trials: Cameron’s Progress With Nusinersen

In this heartwarming video from Robert Harding, we meet Cameron. Cameron was diagnosed with type 1 spinal muscular atrophy (SMA) at five weeks old and was given just six to 12 months to live. The family decided to enroll Cameron in a clinical trial for the drug nusinersen and record his progress.

Find out about the five steps of diagnosing SMA.

The video shows Cameron’s progression and how much he has achieved in his short life. Now at 28 months old, Cameron is able to roll around the floor, dance in his wheelchair, pretend to eat, and has good control of his hands and arms. The family is feeling hopeful about what the drug can continue to do for their son and for other SMA patients.

The drug nusinersen has been developed by Biogen and Ionis Pharmaceuticals and has just had its drug application passed by the Food and Drug Administration (FDA) in America and the European Medicines Agency (EMA). If the drug is approved the company will be able to market the first treatment for SMA using the brand name Spinraza. Find out more about this story here.

In other news for the therapy, Spinraza has met the primary endpoint for a phase 3 clinical trial treating children with later onset SMA (typically Type 2 SMA). It is hoped that the drug will be approved for use in SMA treatment by the end of 2016 or early 2017. Read more about this story here. 

Read about five experimental therapies for SMA you might find interesting. 

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.