Spinal Muscular Atrophy Biomarker Study Reaches Enrollment Goals

We’re excited to announce that enrollment for the NeuroNEXT biomarker study is now fully filled. This means that this critical study can continue to progress and teach us more about finding a cure for SMA.
Cure SMA provided funding for this trial, and we also worked with lead investigator Stephen Kolb, MD, PhD, and his team to recruit families to enroll in this trial.

“We are thrilled to have the support of Cure SMA who are a critical partner in our efforts to inform the SMA community about this study and have provided essential funding support that will have a positive impact on the quality of data that will be generated by this study,” said Dr. Kolb.
Research suggests that treatment for SMA may be most successful if it is delivered very early in life, preferably before six months of age. However, in very young babies, it’s difficult to measure results during the clinical trial to see if the treatment is working.
This study will identify important indicators, called biomarkers, that might help us measure how well a treatment is working in very young babies. The study is looking at these biomarkers in infants with SMA and in healthy infants, so we can compare results. The goal is to gather information that will make future trials more effective.
NeuroNEXT is part of the National Institute of Neurological Disorders and Stroke (NINDS).

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