A higher dose of Spinraza may be more effective than the currently approved dosing schedule for maintaining motor function in ...

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

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 ...

Novartis' OAV101 IT gene therapy showed positive results in Phase 3 trials for SMA, with data supporting its potential as a treatment option.

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 ...

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 ...

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 ...

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 ...