SVB Leerink, for one, predicted last year that 39% of Zolgensma’s $2.7 billion in predicted peak sales would come from the treatment of late-onset SMA in older patients.

Despite an ongoing soap opera surrounding Zolgensma, Novartis’ gene therapy for spinal muscular atrophy (SMA), Wall Street analysts are optimistic about the product overall, with some estimating pe ...

Zolgensma® data shows rapid, significant, clinically meaningful benefit in SMA including prolonged event-free survival, motor milestone achievement and durability now up to 5 years post-dosing ...

"Zolgensma's one-time dose of gene therapy has the potential to make a truly transformative impact on this life-threatening disease," said Kenneth Hobby, president of Cure SMA, a patient advocacy ...

For comparison, Spinraza costs $750,000 in year one, and $375,000 per year thereafter. According to Novartis, Zolgensma's cost is half what it currently costs to treat SMA over a 10-year period.

Zolgensma is currently approved for the treatment of pediatric patients less than 2 years of age with SMA with bi-allelic mutations in the SMN1 gene.

many of parents of children with Spinal Muscular Atrophy (SMA), a rare motor neuron disease. Between one in 6,000 and 10,000 children born have the disorder in some form. More than two-thirds of ...

The only other treatment for SMA is a drug called Spinraza (made by Biogen). But unlike Zolgensma, which is administered just once, Spinraza must be taken for life. The first year of Spinraza costs ...

About Zolgensma Clinical Data The efficacy of Zolgensma in pediatric patients less than 2 years of age with SMA with bi-allelic mutations in the SMN1 gene was evaluated in STR1VE, an open-label ...