RegenXBio will fill its coffers with up to $250 million by monetizing more royalty rights for Novartis-partnered Zolgensma as ...

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 ...

REGENXBIO (NASDAQ:RGNX) announced on Monday the closure of a non-dilutive, limited recourse royalty bond agreement of up to ...

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.

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 ...

Rainbow Children's Hospital in Secunderabad has successfully administered gene therapy to its eighth patient with Spinal ...

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 ...

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).

Devdan Devaraj was diagnosed with Type 2 spinal muscular atrophy when he was just several months old. Read more at ...

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