gene therapy

Bluebird Bio is splitting into two companies, the Wall Street Journal reports.

A gene therapy for Leber hereditary optic neuropathy given to one eye affected vision of the other, according to New Scientist.

Spectrum reports that a clinical trial of a gene therapy for Angelman syndrome is on hold.

Reuters reports that the Food and Drug Administration has rejected BioMarin's gene therapy for hemophilia A and has asked for longer-term data.

A gene therapy for hemophilia could cost millions of dollars, NPR writes.

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.