Infants with SMA Sought for NeuroNEXT Study at UCLA

If you have a young infant with spinal muscular atrophy (SMA), NeuroNEXT could be seeking to talk to you regarding a new study.

NeuroNEXT is an abbreviation for the Network for Excellence in Neuroscience Clinical Trials, and it was created to support efforts by the National Institute of Neurological Disorders and Stroke (NINDS).

NeuroNEXT’s goal: to expand NINDS’s ability “to test promising new therapies, increase the efficiency of clinical trials before embarking on larger studies, and respond quickly as new opportunities arise to test promising treatments for people with neurological disorders,” said Perry Shieh, M.D., Ph.D., associate professor and director of the neuromuscular program at the University of California, Los Angeles (UCLA).

In an announcement to the clinical community, Shieh gave an update on a NeuroNEXT study aiming to “identify biomarkers of disease progression in infants with spinal muscular atrophy.”

The researchers are looking to enroll nine more infants who have SMA and are less than 6 months old.

During the study, the children will be seen by researchers about seven times over two years, with each visit taking place at UCLA and lasting about three hours.

Families of SMA is supporting the project by paying for travel expenses for participants, Shieh added.

UCLA is one of 15 universities and medical centers nationwide involved in the project. Stephen Kolb, M.D., Ph.D., of Ohio State University is the protocol principal investigator of the project.

Noting that there is currently no treatment or cure for SMA and that children with SMA can start showing symptoms within the first few months of life, Kolb said in a news statement, “We are doing this research study because there is strong scientific evidence in animal models of SMA that treatment of SMA may be successful if delivered in the first 3 to 6 months of age in humans and preferably before symptoms develop. At this time, however, researchers who are developing therapies for SMA find that there is little to no information about how to study infants with SMA during the course of a clinical trial. That is why this study is important. The subjects in this study will teach us how to perform the critical SMA therapeutic clinical trials of the future.”

If you would like more information on being considered for the UCLA study, e-mail Angel Hu, the study coordinator, at

For more information on this NeuroNEXT study, including a list of the participating organizations, click HERE.

About the Author

Laurie Watanabe is the editor of Mobility Management. She can be reached at