sickle cell disease

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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.

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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.

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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.

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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.

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Sickle cell gene therapy Casgevy finally cleared for NHS use

Vertex's gene-editing therapy for sickle cell disease, Casgevy, has been given the nod for use by the NHS in England after an earlier rejection.

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...

Sickle cell gene therapy Casgevy finally cleared for NHS use

Chiesi signs $1.9bn deal to buy KalVista and its HAE drug

Using AI-driven synthetic personas to take your insights fur...

sickle cell disease

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