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 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 ...

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

Two patients treated with Zolgensma, a Novartis gene therapy for a rare inherited disorder, have died, highlighting a known but rare side effect risk associated with this and other genetic medicines.

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 ...

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 ...

One of Lorenzo’s action figures sat on a shelf in his room. Novartis has already won coverage for Zolgensma in Russia, Egypt and, most recently, Argentina, where the government announced this ...

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 ...

Novartis (NYSE:NVS) said a Phase 3 study of an intrathecal formulation of its gene therapy Zolgensma, also known as OAV101 IT, met its primary endpoint in the treatment of children with spinal ...