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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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 (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 ...

Novartis’ Zolgensma gene therapy for spinal muscular atrophy is the most expensive drug in the world, but that hasn’t stopped it making a strong start in the US market. Zolgensma (onasemnogene ...

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).

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 ...

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 ...

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 ...

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 ...