"It's encouraging and it gives me hope that they're finally understanding that we deserve to have treatment just like the rest and that we deserve a chance," she said. "At least a ...

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 ...

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 ...

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 ...

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, ...