gene therapy

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.

Nature News reports that gene therapy approaches are tackling sickle cell disease, but that the cost of treatment is a concern.

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

Health insurers in the US are devising new approaches to handle the high costs of gene therapies, the Wall Street Journal reports.

Novartis replaced two top executives from its AveXis unit following data manipulation during early testing of its new gene therapy, Reuters reports.

US Food and Drug Administration says some data from early testing of Novartis' Zolgensma was manipulated, according to Reuters.

Reuters reports that slow adoption of CAR-T therapies might be tied to their high prices.