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tag cure-rare-disease

STAT+: Pharmalittle: Vertex exec says prices for gene therapies are ‘in the ballpark’; insulin access in poor countries remains spotty

STAT

OCTOBER 6, 2022

… Multimillion-dollar prices for a rival’s treatments with the potential to cure rare diseases are “in the right ballpark,” according to Stuart Arbuckle, chief operating officer at Vertex Pharmaceuticals, which is preparing to market its own treatment for sickle-cell disease , Bloomberg News tells us. Even

Drug Pricing

Realising milestones with gene therapy for SMA

European Pharmaceutical Review

MAY 11, 2023

Three SMN-enhancing treatments for the condition are approved by the US Food and Drug Administration (FDA) 2 , however there is no cure for SMA at present. Rare, genetic diseases like SMA are prime targets for gene therapy, but by their very nature, early signs can be difficult to recognise, delaying diagnosis and treatment.

Drug Development

STAT+: Pharmalittle: Vertex exec says prices for gene therapies are ‘in the ballpark’; insulin access in poor countries remains spotty

Realising milestones with gene therapy for SMA

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