Planeta bolilor rare
Dan Alexandru


SMA (spinal muscular atrophy) is a disease that robs people of physical strength by affecting the motor nerve cells in the spinal cord, taking away the ability to walk, eat or breathe. 

There's great reason for hope. Thanks to the dedication of our community and the ingenuity of our researchers we now have the first-ever approved treatment that targets the underlying genetics of SMA. But our work is not done. We know what we need to do to develop and deliver effective therapies. And we're on the verge of further breakthroughs that will continue to change the course of SMA for everyone affected-from infants to adults-and eventually lead to a cure. Let's listen to a patient testimony about life with SMA!