Neuromuscular Disorders

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.


See What We're Doing in Research for Neuromuscular Disorders in Children

Nemours takes great pride in research — so that we can better serve children today and in the future with the most advanced treatments available. One example is Nemours’ Motor Neuron Research Laboratory and Pediatric Engineering Research Lab (or “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 such as muscular dystrophy and spinal muscular atrophy.

Watch a video about one of these devices, called the Wilmington Robotic Exoskeleton (WREX).

Nemours research teams are investigating two promising approaches that may help solve an underlying problem of a neuromuscular disorder called “spinal muscular atrophy” (or “SMA”). Children with SMA lack the ability 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. Many of these studies can be done by taking a skin sample from a child with this condition and monitoring the levels of SMN in these tissues.

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.

Current Clinical Trial Related to SMA:

Mechanisms of Cell Death in Spinal Muscular Atrophy »

Information About Clinical Trials