NVS reports new safety and efficacy data from the phase III program for its investigational OAV101 IT gene therapy for SMA ...

Tenacious efforts at every level, from the individual clinician to the hospital to the state to Congress, will be needed to ...

After bringing Zolgensma to market in 2019 as the first gene therapy for spinal muscular atrophy, Novartis is back with an intrathecal formulation intended for older patients.

The early to mid-stage dose-ranging study evaluated the safety and efficacy of the candidate in patients with SMA with three copies of SMN2 aged 6 months to less than 60 months. The OAV101 IT ...

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 ...

The antisense oligonucleotide works by modulating alternative splicing of the SMN2 exon which is mutated in the disease. This is however "laborious, slow and costly", according to the biopharma.

Since 2016, the FDA has approved three disease-modifying treatments for spinal muscular atrophy, with several ...