Zolgensma works by replacing the missing or nonfunctioning gene causing S.M.A. with a working copy of the gene. It has been given to more than 2,500 children and approved for use in 46 countries.

Zolgensma is a prescription drug that’s used to treat spinal muscular atrophy (SMA) in young children. Zolgensma’s cost may depend on factors such as your child’s treatment plan and whether ...

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Aug 11 (Reuters) - Novartis AG (NOVN.S), opens new tab on Thursday reported two patient fatalities due to acute liver failure following treatment with Zolgensma gene therapy used to treat spinal ...

Novartis has recorded two deaths after treatment with its spinal muscular atrophy gene therapy Zolgensma, once again bringing gene therapy’s safety into attention. Two children in Russia and ...

Zolgensma (onasemnogene abeparvovec-xioi) is a brand-name drug prescribed for spinal muscular atrophy in certain children. Zolgensma comes as a liquid suspension for intravenous (IV) infusion.

A pair of clinical data sets gives Novartis confidence in a new formulation of the company’s gene therapy Zolgensma for the treatment of older patients with spinal muscular atrophy (SMA).

In September 2020, when he was 14 months old, he and his family travelled to the Children’s Hospital of San Antonio in the US where he received his Zolgensma infusion. A year later, he was able ...

Already offering the only non-invasive treatment for SMA in liquid form, Roche’s Evrysdi now comes as a tablet.

REGENXBIO (NASDAQ:RGNX) announced on Monday the closure of a non-dilutive, limited recourse royalty bond agreement of up to $250M with Healthcare Royalty (HCRx). This agreement monetizes select ...

Zolgensma (onasemnogene abeparvovec-xioi) is a prescription gene therapy drug that’s used to treat spinal muscular atrophy in certain children. A healthcare professional will administer the drug ...