Spinal Muscular Atrophy (SMA) causes progressive muscle weakness and, without treatment, can limit life expectancy to just ...

Patients treated with OAV101 IT showed an increase from baseline in HFMSE total score compared with sham control. Topline data were announced from a phase 3 trial evaluating intrathecal onasemnogene ...

"Decreasing AMMO [active maximal mouth opening] over time is seen most often in patients with SMA type 2 with an estimated reduction of almost 1 mm per year...At this point, we propose that ...

In a multidisciplinary follow-up study, researchers analyzed bone health decline and the effects of nusinersen in patients with spinal muscular atrophy (SMA) types 2 and 3. SMA is a progressive, ...

Some patients with later-onset spinal muscular atrophy (SMA) type 2 and type 3 had improved motor function when the investigational monoclonal antibody apitegromab was added to their treatment, the ...

Jesy Nelson has heralded an ‘amazing’ day as newborns to be tested by NHS for spinal muscular atrophy (SMA) - but adds: ‘I ...

Scotland launches a newborn screening pilot for spinal muscular atrophy (SMA) via the heel prick test in Glasgow, aiming for ...

A mother-of-two living with Spinal Muscular Atrophy (SMA) has powerfully declared she "wouldn’t trade my disability," aiming to demonstrate that individuals with disabilities can lead full, meaningful ...