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

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

Speakers from the Muscular Dystrophy Association (MDA) highlighted 75 years of progress in understanding and treating ...

Novartis announces positive data from phase III study of intrathecal onasemnogene abeparvovec in broad patient population with SMA: Basel Friday, March 21, 2025, 11:00 Hrs [IST] N ...

Intrathecal onasemnogene abeparvovec (OAV101 IT), an investigational, one-time gene replacement therapy, demonstrated a ...

treatment with OAV101 IT demonstrated stabilization of motor function over 52 weeks of follow-up in patients who had previously discontinued treatment with Spinraza (nusinersen) or Roche’s RHHBY ...

Novartis NVS reported promising new safety and efficacy results from its phase III studies of investigational intrathecal ...

Novartis has reported positive efficacy and safety outcomes from its Phase III clinical programme for OAV101 IT (onasemnogene ...

Revised: This Reviewed Preprint has been revised by the authors in response to the previous round of peer review; the eLife assessment and the public reviews have been updated where necessary by the ...

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

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