Speakers from the Muscular Dystrophy Association (MDA) highlighted 75 years of progress in understanding and treating ...
Novartis' OAV101 IT gene therapy showed positive results in Phase 3 trials for SMA, with data supporting its potential as a treatment option.
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 ...
A rare genetic condition, Spinal Muscular Atrophy (SMA) affects one in 7,000 people in India. It mostly affects children and ...
Intrathecal onasemnogene abeparvovec (OAV101 IT), an investigational, one-time gene replacement therapy, demonstrated a statistically significant improvement in HFMSE in the Phase III STEER study.
Novartis (NVS) announced positive safety and efficacy results from the Phase III program for investigational intrathecal onasemnogene ...
Phase III Trial Shows Novartis’ Gene Therapy Significantly Improves Spinal Muscular Atrophy Outcomes
The open-label STRENGTH study assessed the safety, tolerability, and efficacy of OAV101 IT in 27 patients with SMA who had previously discontinued nusinersen or risdiplam. Results found that the total ...
This open-label trial assessed the therapy’s efficacy and safety in individuals who had previously been treated with nusinersen or risdiplam. It enrolled 27 subjects with a mean age of 7.4 years.
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