The SMA gene therapy candidate OAV101 IT, a version of Zolgensma, can be safely given to previously treated patients, per new ...

They can switch off genes. Now in occasions with a particularly novel area, you may sometimes be able to amend or alter expression and SPINRAZA is a good experience of using an ASO to alter that ...

Elsewhere in the hospital, an infant — patient No. 1 in a landmark clinical trial — was receiving an IV infusion that ... a chronic treatment called Spinraza. It, too, was pricey, costing ...

Zolgensma is the world’s most expensive drug, with a price tag of more than $2.1 million per one-shot intravenous infusion ... on the market of Biogen’s Spinraza (nusinersen), an antisense ...

at a time when its big selling SMA drug Spinraza (nusinersen) is facing increased competitive pressure. At the same time, Sage has struggled to make headway with its only approved therapy ...

Spinraza (nusinersen), by Biogen ... Administered intravenously (into the vein) through a single infusion, Zolgensma uses a modified and harmless viral shell to deliver a healthy copy of the SMN1 gene ...

Leerink Global Healthcare Conference 2025 March 10, 2025 11:20 AM ETCompany ParticipantsAlisha Alaimo - President of ...