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 ...

Oral function should be measured routinely to determine individual bulbar involvement stages in spinal muscular atrophy (SMA). “Our results underscore the heterogeneity of oral function effort across ...

We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com. Topline results from a phase 3 clinical trial showed an ...

"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 ...

Biomarkers for SMA were identified using LC-MS and machine learning, revealing differences in protein expression across SMA types. CNTN1 and NRXN3 proteins showed potential as prognostic biomarkers, ...

"Our preliminary findings show that German children with SMA, despite significant physical disability, have surprisingly good subjective HRQoL." – Erik Landfeldt, MSc, PhD Previous studies have found ...

When forming a new friendship, columnist Connie Chandler hopes others see her first. But don't ignore the wheelchair entirely ...