Vertex Pharmaceuticals said its medicine could address moderate to severe acute pain, and might be able to avoid the risk of addiction.
Category: Vertex Pharmaceuticals Inc
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New Sickle Cell Therapies Will Be Out of Reach Where They Are Needed Most
There is no clear path for African patients to get access to the treatments, which have multimillion-dollar price tags and are highly complex to manufacture and deliver.
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Panel Tells F.D.A. That CRISPR Sickle Cell Cure Is Safe Enough for Patients
The decision by an advisory committee may lead to Food and Drug Administration approval of the first treatment for humans that uses the CRISPR gene-editing system.
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F.D.A. Experts Will Vote on a Cure for Sickle Cell Disease
The treatment from the company Vertex would be the first medicine to use the gene editing tool CRISPR.
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‘Miracle’ Cystic Fibrosis Drug Kept Out of Reach in Developing Countries
The New York Times – Business:Vertex Pharmaceuticals is not making its drug, Trikafta, available in poorer countries, where thousands of diagnosed patients stand to benefit.