spinal muscular atrophy

In Genome Research this week: transcriptome assembly algorithm, gene expression among somatic motor neurons in spinal muscular atrophy models, and more.

A Minnesota toddler given a gene therapy to treat her spinal muscular atrophy is now walking, according to Newsweek.

The Guardian reports that parents of children with spinal muscular atrophy are concerned about a plan to have a lottery for free gene therapy doses.

Novartis has paused a trial of its Zolgensma gene therapy that treats spinal muscular atrophy, the Wall Street Journal reports.

Parents of children with spinal muscular atrophy tell the Washington Post they are pushing to get insurance coverage of Novartis's Zolgensma.

Novartis's newly approved Zolgensma, a gene therapy for spinal muscular atrophy, is to cost $2.1 million, the AP says.

A second death in gene therapy trial for type 1 spinal muscular atrophy is under investigation, according to Reuters.