Despite significant advancements in spinal muscular atrophy (SMA) management, unmet needs persist, especially for patients ...

Key Findings from the RAG-17 IIT Study Presented at AAN 2025: The final data from the IIT (NCT05903690), a first-in-human, open-label, dose-escalation study involving six SOD1-ALS patients over 240 ...

The following is a summary of “Intrathecal interleukin-6 levels are associated with progressive disease and clinical severity ...

Segmentation Analysis Highlighted with 90 tables and 92 figures, this 179-page report represents a 360-degree view on the global market with extensively detailed segmentations by Offering, Application ...

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 full guideline included 28 recommendations for which there is the greatest evidence and 141 practice points, which reflect the expert opinion of the group. HealthDay News — In a 2024 ...

While the Steer trial enrolled treatment-naïve patients, Novartis also tested the one-time intrathecal gene therapy, coded OAV101 IT, in individuals who had tried but discontinued treatment with ...

The STEER study showed significant motor function improvement in children with SMA using intrathecal onasemnogene abeparvovec. The primary efficacy endpoint was met with a 2.39-point improvement ...

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. Six years ago, the FDA ...