Biogen presented data at the American Academy of Neurology 2019 conference on a potential biomarker for SMA. Biomarkers provide insight on disease progression.
Researchers have found that raised plasma levels of a protein called pNF-H indicate neurological damage and have potential to predict disease 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 Spinraza experienced rapid pNF-H declines followed by stabilisation at lower levels. The results are part of on-going work to identify biomarkers that could provide insight on disease progression in SMA.