Cost-effectiveness body NICE has rejected Biogen’s Spinraza (nusinersen) therapy for the rare genetic muscle wasting disease spinal muscular atrophy (SMA) because of its high cost – but is in ...

Treatment with Spinraza (nusinersen) can help stabilize or improve motor function in adults with SMA, according to a new ...

In the US, a single shot of Zolgensma (onasemnogene abeparvovec) costs $2.1 million, while Spinraza (nusinersen) costs $750,000 in its first year. Biogen said it aims to begin a phase IV study as ...

Amino acid balance could play a role in SMA, as a study showed acids able to influence nervous system function were ...

Each take home $3 million. Molecular geneticist Adrian Krainer of Cold Spring Harbor Laboratory received the award for his part in developing nusinersen (Spinraza), an oligonucleotide therapeutic for ...

This is in contrast to the approved 12 mg regimen of SPINRAZA, the brand name under which nusinersen is currently commercialized in over 71 countries. The applications for the updated dosing ...

tolerability and efficacy of OAV101 IT in patients with SMA aged two to less than 18 years who had discontinued treatment with nusinersen or risdiplam. Key findings: OAV101 IT demonstrated a ...

Nusinersen is currently commercialized under the brand name SPINRAZA in over 71 countries at the label-approved dose of 12 mg. “Continued progress to improve upon the remarkable initial ...

The STRENGTH study found OAV101 IT stabilized motor function in patients who had discontinued Spinraza or Evrysdi ... who had discontinued treatment with nusinersen or risdiplam.