Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...

Panelists discuss how spinal muscular atrophy is an autosomal recessive genetic disease affecting motor neurons with 3 currently approved disease-modifying therapies that restore SMN protein ...

A positive newborn screening for spinal muscular atrophy (SMA) is currently considered a medical emergency. Without early treatment, severe disability or death in infancy are likely. However, research ...

Novartis (NVS) on Thursday announced that the European Commission approved its gene therapy. Itvisma (onasemnogene abeparvovec) for children aged two years and older with spinal muscular atrophy, a ...

Jason is a general assignment reporter, with particular focus on genetic medicine and rare disease. Confidential tips can be sent on Signal at JasonMast.77. Nearly six years ago, the Food and Drug ...

Itvisma should only be administered intrathecally using a lumbar puncture by health care professionals experienced in performing the procedure. The Food and Drug Administration has approved Itvisma ® ...

Tripura Chief Minister, Manik Saha, has directed officials to extend all possible assistance for the advanced treatment of a ...

Although a profound transformation has occurred in both survival and clinical course, long-term data on the efficacy and safety of disease-modifying therapies is required to inform their use in ...

Megan covers the intersection of science and society. How is biomedical research funded? Who benefits? And how do new technologies reflect and shape our values? You can reach Megan on Signal at ...

Former Little Mix star Jesy Nelson has said she's been reduced to tears by her children being forced to wear spinal jackets ...

The teen's mother said he first began showing symptoms at 2 months old when “his arms and legs were just hanging by his side” ...