Nature Cell Biology - Spinal muscular atrophy is caused by mutations in the survival motor neurons gene, SMN1. The SMN protein localizes to both the cytoplasm and the nuclear bodies. One hallmark ...

The scientists at the University of California, San Francisco, and Johns Hopkins University in Baltimore injected an ...

Watch Video Spinal muscular atrophy (SMA) describes a group of disorders associated with spinal motor neuron loss. The different forms of SMA are associated with numerous gene mutations and ...

In a hopeful step for medicine, a 2-and-a-half-year-old child born with spinal muscular atrophy (SMA ... induce increased ...

Injecting medicine into the amniotic fluid staves off progression of spinal muscular atrophy in utero.Evidence is mounting ...

The therapy used molecules called antisense oligonucleotides, or ASOs, that can alter the expression of genes through interactions with RNA, which creates proteins. ASOs are currently given to babies ...

A wearable robotic device called HAL improved walking ability for SMA patients who also received Spinraza (nusinersen), a ...

This is the first time spinal muscular atrophy (SMA), a severe motor ... which is responsible for producing the survival ...

Roche is hoping to undercut hugely expensive rivals after the FDA approved its oral spinal muscular atrophy ... production of the survival of the motor neuron (SMN) protein.

Digital PCR to simultaneously detect SMN1 gene defects and the number of SMN2 copies to diagnose SMA matches standard methods ...