Spinal Muscular Atrophy (SMA)

At Nemours, we promise to do whatever it takes to treat children as we would our own. When your child comes to Nemours, we know you’re placing your trust in us. This trust and our dedication to improving the health of your child is what inspires us to provide exceptional care and the most satisfying experience possible.


What We're Doing in Spinal Muscular Atrophy Research

Nemours takes great pride in research — so that we can better serve children today and in the future with the most advanced treatments available. Nemours research teams are investigating two promising approaches that may help solve an underlying problem of SMA — an inability to make a protein called SMN, which protects the motor neurons that tell muscles what to do. Without this important protein, the motor neurons are damaged, along with many of the body’s core muscles.

In one approach, we’re looking at ways to induce the body to make more SMN protein. In a second approach, we’re investigating if there’s a way to keep motor neurons alive even if there’s not enough SMN protein to protect them.

We often offer clinical trials related to spinal muscular atrophy. A clinical trial is a study that evaluates new medicines, new devices, new treatments or new applications for old treatments. Clinical trials are safe and supervised by a qualified physician, under strict guidance of the Food and Drug Administration (FDA). Patient safety is extremely important to the FDA, to those conducting the studies, and to everyone at Nemours. In a clinical trial, children participating in the study are monitored closely — and families may refuse any aspect of the study treatment or completely discontinue their child’s participation in the clinical trial at any time.

Another example is Nemours’ Pediatric Engineering Research Lab (PERL), part of the Center for Orthopedics Research and Development (CORD). Here, our researchers are responsible for developing amazing new technologies, devices and therapies that can dramatically change the lives of kids with neuromuscular disorders, including SMA. Watch a video about one of these devices, called the Wilmington Robotic EXoskeleton (WREX).

Clinical Trials Related to SMA