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New lab test may help in studying effects of Spinraza treatment

Researchers have developed a new laboratory technique for measuring levels of Spinraza (nusinersen), an approved treatment for spinal muscular atrophy (SMA), in biological fluids. The test is expected to help overcome some limitations of other approaches now in use, and may therefore aid scientists and physicians when studying the…

Abnormal immune system activation seen in SMA fly models

A range of genetic mutations associated with spinal muscular atrophy (SMA) were linked to abnormal activation of the immune system in fly models, which a team of U.S. researchers believe could be a mechanism that contributes to neurodegeneration in the rare disease. Altogether, the evidence indicated that these…