SMA 'NeuroNEXT' Biomarkers Study Seeks Participants

An informational video describes an ongoing National Institutes of Health study of natural history and biomarkers in spinal muscular atrophy

Article Highlights:
  • Researchers studying natural history (disease course) and biomarkers (biological indicators of disease) in infants with spinal muscular atrophy are recruiting SMA-affected and healthy infants in an effort to detect differences in how children develop.
  • Investigators hope to enroll as many as 54 infants (27 with SMA and 27 unaffected) in the study, which will be conducted at 15 sites around the country.
  • The study seeks to determine the most effective time frame for administering SMA treatments, and how to study infants with SMA in any future clinical trials of experimental SMA therapies.
  • A video featuring a neurologist and a parent of a child with SMA describes study details.
by Amy Madsen on February 13, 2013 - 9:41am

Update (April 2, 2014): As of March 31, 2014, the investigators were seeking four SMA-affected infants.

Update (Jan. 9, 2014): As of Dec. 30, 2013, the investigators were seeking eight SMA-affected infants.

Update (Nov. 4, 2013): As of Oct. 21, 2013, this study had finished its recruitment of 27 infants without SMA and had enrolled 12 infants with SMA. As of that date, the investigators were seeking 15 additional infants with SMA.

Update (March 28, 2013): This story was updated with a link to the site for this study.

Researchers conducting a study of natural history and biomarkers in infants with type 1 spinal muscular atrophy (SMA) currently are recruiting participants at 15 locations across the United States.  

The National Institute of Neurological Disorders and Stroke (NINDS), part of the U.S. National Institutes of Health, is conducting the study through its Network for Excellence in Neuroscience Clinical Trials (NeuroNext). Twelve of the 15 sites are at MDA clinics.

Reliable biomarkers (biological indicators) for SMA could allow doctors or researchers to evaluate disease progression and determine whether an experimental treatment is working or not.

The study also seeks to determine when treatments in SMA should be given to be most effective, and how to best study infants with SMA during the course of future clinical trials designed to test SMA therapies.

Study information

To participate in the study, called Spinal Muscular Atrophy Biomarkers in the Immediate Postnatal Period of Development, infants must be between 0 and 6 months of age at the time of enrollment.

Study participants are required to make regular visits to the study site at ages 1 month, 3 months, 6 months, 9 month, 12 months, 18 months and 24 months. At these visits, researchers will observe each infant's growth and development, using a variety of procedures that include:

  • motor function tests;
  • noninvasive tests to measure nerve and muscle function; and
  • a blood draw (performed on most but not all visits).

Participants may be paid $50 for each visit and receive reimbursement for travel costs.

For more information

To spread the word about the study, NINDS has created a video featuring a researcher and a parent of a child with SMA that explains what is known about SMA and the goals of the study.

Information also is available in the study brochure and the study website.

For answers to general questions about participation in the study, email, or call (855) SMA-BIOM (855-762-2466). You also can view Spinal Muscular Atrophy (SMA) Biomarkers in the Immediate Postnatal Period of Development, or enter NCT01736553 into the search box at



Boston Children’s Hospital, Boston, Mass.

Children’s National Medical Center, Washington, D.C.

Columbia University Medical Center, New York, N.Y.

State University of New York Upstate Medical Center, Syracuse, N.Y.


Northwestern University, Chicago, Ill.

Ohio State University, Columbus, Ohio (site of the protocol principal investigator)

Washington University in St. Louis School of Medicine, St. Louis


Emory University, Atlanta, Ga.

  • Children’s Hospital of Atlanta
  • Principal Investigator: Maurice Sholas
  • Coordinator: Rian Thornton
  • Email:

University of Texas Southwestern Medical Center, Dallas, Texas

Vanderbilt University, Nashville


University of California, Davis

University of California, Los Angeles

University of Colorado, Denver, Aurora, Colo.

University of Utah, Salt Lake City

Oregon Health and Science University, Portland, Ore.

  • Dorenbecher Children’s Hospital, Portland
  • Principal Investigator: Erika Finanger
  • Coordinator: Rhonda Muhly
  • Email:
About Clinical Trials

About Clinical Trials

A clinical trial is a test, in humans, of an experimental treatment. Although it's possible that benefit may be derived from participating in a clinical trial, it's also possible that no benefit, or even harm, may occur.

MDA has no ability to influence who is chosen to participate in a clinical trial.

To learn more, see Learn About Clinical Studies and Being a Co-Adventurer, which is about neuromuscular disease clinical trials. To see a continuously updated database of clinical trials, go to

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