The RESPOND study will seek to understand if the proven efficacy of SPINRAZA and its mechanism of action, which leads ... patients previously treated with Zolgensma to further optimize treatment ...

These human data support the mechanism of action initially identified in non-human non-clinical studies in murine and non-human primate models, that a single intravenous administration of Zolgensma is ...

The FDA has said Novartis could face legal action, after it emerged that data filed to support approval of its spinal muscular atrophy gene therapy Zolgensma had been manipulated. The US regulator ...

The FDA revealed earlier this month that it may take action against Novartis after discovering that data filed to support approval of Zolgensma (onasemnogene abeparvovec) – a spinal muscular ...

Novartis already markets an IV-administered version of the gene therapy called Zolgensma.The product, which is one of the ...

Novartis has acknowledged that two patients have died of acute liver failure following treatment with its Zolgensma® (onasemnogene ... step in markets where this action is supported by health ...

The crown jewel of that deal is Zolgensma, a gene therapy to treat the neuromuscular disorder spinal muscular atrophy, or SMA. It won approval from the Food and Drug Administration in 2019.

Basel, March 4, 2024 – Novartis today presented new data that continue to support the clinical benefits of Zolgensma ® (onasemnogene abeparvovec), the only one-time gene therapy for the ...

Novartis AG said a new experimental medicine improved motor function in children with a form of spinal muscular atrophy, the devastating disorder targeted by its gene therapy Zolgensma ...

Two children taking the gene therapy drug onasemnogene abeparvovec (Zolgensma, Novartis ... as an additional step in markets where this action is supported by health authorities," the ...

One of two children who died after being treated with Novartis AG’s Zolgensma was past the US age limit for receiving the gene therapy for spinal muscular atrophy, suggesting the patient was at ...

The RESPOND study will seek to understand if the proven efficacy of SPINRAZA and its mechanism of action, which leads ... patients previously treated with Zolgensma to further optimize treatment ...