The American Journal of Managed Care

Phase 3 STEER Study of SMA Gene Therapy Meets Primary End Point

Intrathecal onasemnogene abeparvovec improved HFMSE scores in patients with SMA type 2, indicating enhanced motor function. The STEER study confirmed the therapy's favorable safety profile, with ...
The American Journal of Managed Care

Splicing Modifiers and Gene Therapy for SMA

Panelists discuss how splice modifiers work by enhancing protein production from the SMN2 gene, with risdiplam (Evrysdi) being an oral daily medication and nusinersen (Spinraza) being administered ...
Medscape

Gene Therapy Tops Nusinersen in Spinal Muscular Atrophy

Treatment with onasemnogene abeparvovec gene therapy for spinal muscular atrophy (SMA) type 1 was associated with less need for nutritional support or nighttime ventilation compared to recommended ...
Yahoo

Novartis wins approval to use SMA gene therapy in older patients

Add Yahoo as a preferred source to see more of our stories on Google. The Food and Drug Administration on Monday approved a new version of Novartis’ gene therapy for spinal muscular atrophy, aimed at ...
Hosted on MSN

FDA approves Novartis’ Itvisma, first gene therapy for SMA patients 2 and older

Novartis (NVS) on Monday said that the U.S. FDA has approved Itvisma for the treatment of children two years and older, teens, and adults living with spinal muscular atrophy with a confirmed mutation ...
NJBIZ

FDA approves Novartis’ Itvisma gene therapy for SMA

Novartis has received approval from the U.S. Food and Drug Administration for Itvisma, a treatment for spinal muscular atrophy. Dr. John Day is professor of neurology and pediatrics, director of the ...
FiercePharma

Novartis scores FDA approval for new version of SMA gene therapy, prices at $2.59M

Novartis has received an FDA approval for Itvisma, a new version of the company’s gene therapy Zolgensma, to treat older patients with spinal muscular atrophy (SMA). The approval comes more than six ...
Healio

Gene therapy slows progression of spinal muscular atrophy type 2

We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com. Topline results from a phase 3 clinical trial showed an ...
MarketWatch

Novartis receives FDA approval for Itvisma(R), the only gene replacement therapy for children two years and older, teens, and adults with spinal muscular atrophy (SMA)

The MarketWatch News Department was not involved in the creation of this content. -- Itvisma (onasemnogene abeparvovec-brve) demonstrated improved motor function and stabilization in patients ...
Medical Xpress

Gene therapy improves movement in kids with spinal muscular atrophy

A single-dose gene replacement therapy is found to improve movement ability in children over 2 years of age and teenagers with spinal muscular atrophy, according to research published in Nature ...
Hosted on MSN

Novartis gene therapy Itvisma approved in EU for spinal muscular atrophy

Novartis (NVS) on Thursday announced that the European Commission approved its gene therapy. Itvisma (onasemnogene abeparvovec) for children aged two years and older with spinal muscular atrophy, a ...
Benzinga.com

FDA OKs Novartis SMA Treatment As First Gene Therapy Option For Kids, Teens And Adults

On Monday, the U.S. Food and Drug Administration (FDA) approved Novartis AG’s (NYSE: NVS) Itvisma (onasemnogene abeparvovec-brve) for children two years and older, teens, and adults with spinal ...