The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.

  1. Potential biomarker for SMA identified

    Potential biomarker for SMA identifiedBiogen 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.

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