STAT News

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

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Gene Therapy Gene Insulin Drugs 98

XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. Casgevy is also the first ever CRISPR/Cas9-based therapy approved in the US.

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Gene Therapy FDA Approval Gene In-Vivo 98

The Pharma Data

OCTOBER 21, 2020

21, 2020 — A combination of two “targeted” therapies can beat back a rare form of blood cancer — without the toxic effects of chemotherapy, a new study has found. Experts said it is too soon to call the approach a “cure.” It’s too soon to say this is a cure.” WEDNESDAY, Oct.

Drugs 52

Drugs Genetics Trials Research 52

The Pharma Data

OCTOBER 21, 2020

21, 2020 — A combination of two “targeted” therapies can beat back a rare form of blood cancer — without the toxic effects of chemotherapy, a new study has found. Experts said it is too soon to call the approach a “cure.” It’s too soon to say this is a cure.” WEDNESDAY, Oct.

Drugs 52

Drugs Genetics Trials Research 52