Skip to main content
Premium Trial:

Request an Annual Quote

Pricey, But Available

A pricey gene therapy treatment for spinal muscular atrophy (SMA) will be available through the National Health Service in England later this year, the Guardian reports.

SMA, a genetic disorder affecting infants and children, leads to paralysis, breathing problems, and death, Novartis's Zolgensma delivers a working copy of the SMN gene to halt disease progression. The gene therapy was approved in the US in 2019 to treat children under the age of two with certain forms of the disease, as indicated by genetic testing. At the time, the Associated Press noted that the gene therapy was the most expensive medicine at a list price of $2.1 million.

According to the Guardian, the NHS says it has negotiated a discount with the drugmaker, though it is still the most expensive treatment approved by the National Institute for Health and Care Excellence. "SMA is the leading genetic cause of death among babies and young children, which is why NHS England has moved mountains to make this treatment available, while successfully negotiating hard behind the scenes to ensure a price that is fair to taxpayers," Simon Stevens, the chief executive of NHS England, tells the Guardian

It adds that about 65 babies in England each year are born with SMA.

The Scan

Latent HIV Found in White Blood Cells of Individuals on Long-Term Treatments

Researchers in Nature Microbiology find HIV genetic material in monocyte white blood cells and in macrophages that differentiated from them in individuals on HIV-suppressive treatment.

Seagull Microbiome Altered by Microplastic Exposure

The overall diversity and the composition at gut microbiome sites appear to coincide with microplastic exposure and ingestion in two wild bird species, according to a new Nature Ecology and Evolution study.

Study Traces Bladder Cancer Risk Contributors in Organ Transplant Recipients

In eLife, genome and transcriptome sequencing reveal mutation signatures, recurrent somatic mutations, and risky virus sequences in bladder cancers occurring in transplant recipients.

Genes Linked to White-Tailed Jackrabbits' Winter Coat Color Change

Climate change, the researchers noted in Science, may lead to camouflage mismatch and increase predation of white-tailed jackrabbits.