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Double gene therapy leads to lasting benefits in SMA mouse model

A combination genetic therapy approach designed to restore more normal activity of the SMN1 gene improved motor function and prolonged survival in a spinal muscular atrophy (SMA) mouse model. Called Gene-DUET, it involves supplementing the body with additional healthy SMN1 genetic material, an approach similar to the approved gene therapy…

SMA prevalence at birth lower than thought, US study says

The prevalence of spinal muscular atrophy (SMA) at birth in the U.S. is lower than the historic global SMA birth prevalence estimate, according to data from newborn screening programs from 30 U.S. states. The findings were published as a research letter, “Newborn Screening and Birth Prevalence for Spinal…

Inflammatory signaling molecules may predict response to Spinraza

In people with spinal muscular atrophy (SMA) treated with Spinraza (nusinersen), levels of pro-inflammatory signaling molecules tend to decrease while levels of anti-inflammatory signaling molecules tend to increase in the months after starting treatment, a study has found. Findings also suggested that changes in certain inflammation-regulating molecules may…