Tag Archives: Price

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

Bluebird Bio CEO Nick Leschly on Friday defended the biotech company’s $ 1.8 million price tag for its new gene therapy to treat a rare genetic blood disorder. Bluebird’s therapy, Zynteglo, was approved in Europe earlier this month for patients with beta thalassemia who require regular blood transfusions to manage their disease and have no… Read More »

The most expensive medicine ever treats a rare condition. The price: $2.13 million – Los Angeles Times

Evelyn Villarreal was one of the first children treated, at 8 weeks. Her family, from Centreville, Va., had lost their first child to spinal muscular atrophy at 15 months. Two years later when Evelyn was born a test showed she also had the disease, so the family enrolled her in the gene therapy study at… Read More »

Drug price hearing on Capitol Hill has similarities to one nearly 60 years ago

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