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

Switching to Gene Therapy After Nusinersen or Risdiplam May Benefit Patients With SMA

Onasemnogene abeparvovec gene therapy post-nusinersen or risdiplam showed meaningful motor improvements in children with SMA, with a manageable safety profile. The study cohort included older, heavier ...
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 ...
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 ...
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 ...
Healio

Gene therapy improves motor function in spinal muscular atrophy at 52 weeks

We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com. Patients treated with onasemnogene abeparvovec recorded ...
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 ...
Monthly Prescribing Reference

One-Time Gene Therapy Itvisma Approved for Spinal Muscular Atrophy

Credit: Novartis. The approval was supported by data from phase 3 STEER study, which enrolled patients with SMA who were treatment-naive and able to sit but never able to walk independently. Itvisma ...
Nasdaq

Novartis' Gene Replacement Therapy For SMA Demonstrates Meaningful Efficacy And Safety Results

(RTTNews) - Novartis AG (NVS), Wednesday, announced the positive results from its Phase III clinical program of investigational gene replacement therapy, intrathecal onasemnogene abeparvovec or OAV101 ...
WebMD

Itvisma: FDA Approves One-Time Gene Therapy for Spinal Muscular Atrophy in Children 2 or Older, Teens, and Adults

What Is Itvisma, and Why Does It Matter? Itvisma (onasemnogene abeparvovec-brve) is a gene therapy that the FDA has approved to treat spinal muscular atrophy (SMA) in adults, teens, and children 2 or ...
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 ...