Skip to main content

Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

Gene therapy rescues newborns with spinal muscular atrophy

Nature Biotechnology volume 36, pages 669–670 (2018)Cite this article

Newborn screening for spinal muscular atrophy (SMA), the most lethal inherited condition in infants, has begun in several US states, just as the disease was included in an official list of genetic diseases recommended for routine testing. The decision taken by the Department of Health and Human Services on July 3 to add SMA to the Recommended Uniform Screening Panel (RUSP) is an important milestone, given that an approved drug Spinraza (nusinersen) is available and several others are likely to gain approval in the near future, including what appears to be a highly effective gene therapy (Table 1). As Nature Biotechnology went to press, four states—Indiana, Minnesota, Missouri and Utah—had already included SMA in their neonatal screening programs, and five more—Georgia, Massachusetts, New York, North Carolina and Wisconsin—had begun or were about to start SMA screening pilots, according to the patient advocacy group Cure SMA. As the management of SMA enters a new era, the question is whether gene therapy will make small-molecule and antisense drugs redundant.

Table 1 Selected SMA drug development programs
Full size table

This is a preview of subscription content, access via your institution

Access options

Buy article

Get time limited or full article access on ReadCube.

$32.00

Buy

All prices are NET prices.

Author information

Authors and Affiliations

  1. Dublin

    Cormac Sheridan

Authors
  1. Cormac Sheridan
    View author publications

    You can also search for this author in PubMed Google Scholar

Rights and permissions

Reprints and Permissions

About this article

Verify currency and authenticity via CrossMark

Cite this article

Sheridan, C. Gene therapy rescues newborns with spinal muscular atrophy. Nat Biotechnol 36, 669–670 (2018). https://doi.org/10.1038/nbt0818-669

Download citation

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1038/nbt0818-669

This article is cited by

Search

Advanced search

Quick links

Nature Briefing

Sign up for the Nature Briefing newsletter — what matters in science, free to your inbox daily.

Get the most important science stories of the day, free in your inbox. Sign up for Nature Briefing