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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 announced positive safety and efficacy results from the phase III programme for investigational intrathecal onasemnogene abeparvovec (OAV101 IT) in a broad population of patients aged two to ...

Novartis' OAV101 IT showed a 2.39-point HFMSE improvement in SMA patients vs. 0.51 points in the sham control group. The STRENGTH study found OAV101 IT stabilized motor function in patients who ...

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 ...

Nirmatrelvir-ritonavir was beneficial for COVID-19 management in patients with chronic kidney disease or kidney failure, significantly reducing the risk of mortality and hospital admission. Paxlovid ® ...