sickle cell disease

News

Vertex gets paediatric FDA okay for Casgevy gene therapy

For the first time, children in the US as young as two with blood disorders sickle cell disease and beta thalassaemia are eligible for gene therapy.

News

Novo Nordisk preps filings for sickle cell drug etavopivat

Novo Nordisk has claimed a key win in its head-to-head race with Agios to bring an oral PKR activator to the market for sickle cell disease.

News

Agios' chief medical officer, Sarah Gheuens

Agios mulls early FDA filing for sickle cell therapy

Agios is planning to file its sickle cell disease candidate mitapivat with the FDA under the accelerated pathway within the next few months.

News

Agios shares slump on sickle cell disease data

Shares in Agios lost nearly half their value after it posted mixed clinical results from a phase 3 trial of its sickle cell disease therapy mitapivat.

News

Pfizer suffers another hit to its GBT sickle cell portfolio

Pfizer's $5.4 billion takeover of Global Blood Therapeutics three years ago is starting to look like a poor deal.

Digital

Enhancing rare disease trials: eCOA technology’s impact on s...

Sickle cell disease (SCD) affects an estimated 20 million people worldwide.

Vertex gets paediatric FDA okay for Casgevy gene therapy

Novo Nordisk preps filings for sickle cell drug etavopivat

Agios mulls early FDA filing for sickle cell therapy

Agios shares slump on sickle cell disease data

Pfizer suffers another hit to its GBT sickle cell portfolio

Enhancing rare disease trials: eCOA technology’s impact on s...

Lundbeck preps for key bexicaserin data around year-end

Discover a new era of healthcare panel engagement: Grounded ...

sickle cell disease

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