Potential Biomarker for SMA Identified

10 May 2019

Biogen presented data at the American Academy of Neurology 2019 conference on a potential biomarker for SMA. 

Biomarkers provide insight on condition progression, and can be a valuable tool in determining the effectiveness of drugs being tested in clinical trials.  Researchers have found that raised levels of a protein called pNF-H in blood plasma indicate neurological damage and have potential to predict condition activity. The advantage of pNF-H is that it is present in blood and therefore easily measured. 

The scientists showed that the level of pNF-H was higher in individuals with SMA than those without SMA. Individuals from the NURTURE, ENDEAR and CHERISH studies treated with SpinrazaTM experienced rapid pNF-H declines followed by stabilisation at lower levels, indicating that pNF-H can correlate with condition severity and progression.

pNF-H is raised in several other nervous system conditions, and is therefore not specific to SMA. Nevertheless, pNF-H is likely to be a valuable biomarker for SMA, and the presented results are part of on-going work to identify biomarkers that could provide insight on condition progression in SMA. 

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