Novartis: promising results for SMA treatment

(CercleFinance.com) - Novartis announces positive results from a Phase III program with the intrathecal onasemnogen abeparvovec (OAV101 IT) in patients aged 2 years to less than 18 years with spinal muscular atrophy (SMA).


OAV101 IT is presented as an investigational gene therapy that treats the genetic cause of SMA with a single dose, and offers a favorable safety profile.

Specifically, the study showed a significant improvement of 2.39 points on the HFMSE scale for OAV101 IT, compared with 0.51 point in the control group. In addition, OAV101 IT stabilised motor function in patients who had discontinued nusinersen or risdiplam. In addition, no major complications were observed, and the safety profile is considered favorable.

These results will be presented at the MDA conference in Dallas in March 2025.


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