Intrathecal onasemnogene abeparvovec improved HFMSE scores in patients with SMA type 2, indicating enhanced motor function. The STEER study confirmed the therapy's favorable safety profile, with ...

"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, ...

We were unable to process your request. Please try again later. If you continue to have this issue please contact [email protected]. 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 ...

Please provide your email address to receive an email when new articles are posted on . At the time of last follow up, 82.6% of children born with SMA type 1 from 2016 on were still alive. Of the 140 ...

MIDWAY, Tenn. (WJHL)- 10-year-old Dagan McMahan has Spinal Muscular Atrophy (SMA) Type 2, a degenerative disease that weakens the muscle in his body. Dagan is unable to walk or stand; he gets around ...

Spinal muscular atrophy (SMA) type 1 is a genetic condition in which the nerves that control movement, breathing, and swallowing become progressively weaker and smaller. SMA has five types with ...

Scholar Rock's lead candidate, apitegromab, showed promising Phase 3 results in SMA, boosting shares by over 325%, with plans for regulatory submissions in the US and EU. Despite positive data, ...