A mom was overjoyed when she learned a drug could save her baby's life. Then she learned it cost $2 million.

A mom was overjoyed when she learned a gene therapy treatment could save her child's life, until she found out no one would cover the $2 million price tag.
Medscape

Gene Therapy Improves Movement in Older Children With SMA

Determining which patients should receive the treatment will still require individual risk-benefit assessments, experts say.
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 ...

FDA approves Itvisma for spinal muscular atrophy

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

New gene therapy drugs can save lives, but many cost millions

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 ...
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 ...
EurekAlert!

Researchers evaluate the benefit of dual therapy for children at risk for spinal muscular atrophy

In a first-of-its-kind study, researchers compared the efficacy of preventative therapy for spinal muscular atrophy (SMA) between two well-matched study groups, using either gene therapy (onasemnogene ...
Rheumatology Advisor

One-Time Gene Therapy Itvisma Approved for Spinal Muscular Atrophy

The FDA approved Itvisma gene therapy for patients aged 2 years and older with spinal muscular atrophy and confirmed SMN1 gene mutation.
equities

Genentech’s Spinal Muscular Atrophy Therapy Shows Significant Improvement in Motor Function

Spinal muscular atrophy (SMA) is a progressive genetic disease that causes devastating muscle atrophy and disease-related complications. SMA is the leading genetic cause of death in infants and ...
abc27

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

EAST HANOVER, N.J., Nov. 24, 2025 /PRNewswire/ -- Novartis today announced that the US Food and Drug Administration (FDA) has approved Itvisma ® (onasemnogene abeparvovec-brve) for the treatment of ...