Despite significant advancements in spinal muscular atrophy (SMA) management, unmet needs persist, especially for patients ...

A case report in Japan shows Spinraza helped a boy with SMA type 0 sit without support for a short time at age 2 and stand ...

Spinal muscular atrophy (SMA) treatment has evolved significantly ... I would much rather be treated with nusinersen, where I know that I can come in quarterly and have a spinal tap and get ...

Scholar Rock tested apitegromab in patients aged two to 12 who were already being treated with the standard therapies for SMA, Biogen's antisense-based therapy Spinraza (nusinersen) and Roche's ...

Biogen Inc. (NASDAQ:BIIB), which licensed the rights to nusinersen from Ionis ... has been a foundational treatment in SMA, ...

A pioneering team at St Jude Children's Research Hospital in the US say they successfully treated a child diagnosed with SMA while still ... oligonucleotide Spinraza (nusinersen) – which has ...

Three men with late-onset spinal muscular atrophy (SMA) on Evrysdi treatment were able to conceive with their partners, per a ...

In this trial, the efficacy and safety of the therapy were evaluated against a sham control in treatment-naïve SMA Type 2 subjects ... had previously been treated with nusinersen or risdiplam.

Results found that patients treated with OAV101 IT experienced a 2.39 ... and efficacy of OAV101 IT in 27 patients with SMA who had previously discontinued nusinersen or risdiplam. Results found that ...

The higher dose regimen of nusinersen comprises a more ... As a foundation of care in SMA, more than 14,000 individuals have been treated with SPINRAZA worldwide.1 The currently approved 12 ...