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 ...

The U.S. Food and Drug Administration has approved Itvisma (onasemnogene abeparvovec-brve) for the treatment of spinal ...

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.

Novartis secured approval for its drug to treat certain patients diagnosed with spinal muscular atrophy from the Food and Drug Administration. Itvisma, or onasemnogene abeparvovec-brve, was approved ...

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 ...

The effects allowed these minors who could sit but not stand to move like they've never done before, including walking and ...

The FDA has approved an adeno-associated virus 9 gene replacement therapy to treat individuals aged 2 years and older with spinal muscular atrophy and a confirmed mutation of the survival motor neuron ...

A new class of life-saving drugs is helping children who once had no hope. But some carry a price tag of millions for a ...

Loss of muscle mass, also called muscle wasting or atrophy, can happen gradually as part of getting older or more suddenly ...

Two children with a rare genetic condition will live very different lives because one was tested at birth and the other was not, their parents say. Marley, five, and Meadow, four months, both have ...

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 ...