Novartis AG (NYSE:NVS) announced safety and efficacy results from the Phase 3 program for investigational intrathecal ...

Novartis (NVS) announced positive safety and efficacy results from the Phase III program for investigational intrathecal onasemnogene ...

The first prenatal therapy for spinal muscular atrophy (SMA) showed promising ... It's one of three drugs approved for SMA, the others being nusinersen (Spinraza) and onasemnogene abeparvovec ...

Scholar Rock , a late-stage biopharmaceutical company focused on advancing innovative treatments for neuromuscular diseases, cardiometabolic disorders, and other serious diseases where protein growth ...

Roche is hoping to undercut hugely expensive rivals after the FDA approved its oral spinal muscular atrophy (SMA ... after Biogen’s Spinraza (nusinersen) and Novartis’ Zolgensma (onasemnogene ...

It is the first muscle-targeted treatment candidate in spinal muscular atrophy (SMA ... were receiving current standard of care (either nusinersen or risdiplam). SAPPHIRE enrolled 156 patients ...

Fostering dialogue between neurologists, researchers, patient representatives, and policymakers is an important step towards ...

NfL levels have proven valuable in monitoring treatment responses in spinal muscular atrophy, which causes ... with the antisense oligonucleotide nusinersen, NfL levels in CSF were normalized ...

Guselkumab for previously treated moderately to severely active Crohn's disease ID6238 Technology appraisal guidance TBC Nusinersen and risdiplam for treating spinal muscular atrophy (review of TA588 ...

Shares in Scholar Rock have gone into overdrive after the biotech reported positive phase 3 results with apitegromab in patients with spinal muscular atrophy (SMA), setting up regulatory filings ...