Bluebird CEO defends $1.8 million gene therapy price — ‘It’s really thinking about it differently’

Bluebird Bio's $ 1.8 million gene therapy, Zynteglo, is the second most expensive drug in the world behind Novartis' $ 2.1 million gene therapy for spinal muscular atrophy. Health and Science U.S.HEALTHCARE UPDATE: Click today to request your free ACRX … Read more…

Commentary: Novartis CEO: Gene therapy offers hope of cures in a treatment, but US needs new pricing, payment model

The current global health system treats chronic diseases with a pay-as-you-go model, spreading costs over months and years. It's unprepared to pay for a surge of new, single-treatment therapies with the potential to provide a lifetime of benefit. Health and … Read more…

Brain responses to language in toddlers with Autism linked to altered gene expression

Scientists have identified a previously unknown, large-scale association between molecular gene expression activity in blood leukocyte cells and altered neural responses to speech in toddlers with autism as measured with functional magnetic resonance imaging. Child Development News — ScienceDaily SPECIAL … Read more…

Gene variants raise risk of migraines in African-American children

Researchers have discovered common gene variants associated with migraines in African-American children. The research adds to knowledge of genetic influences on childhood migraine and may lead to future precision medicine treatments for African-American children with these intense headaches. Child Development … Read more…