Remove tag blood-clots
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Grand Rounds February 16, 2024: Clinical Implications of the MINT Trial: p=0.07 (Jeffrey Carson, MD, MACP)

Rethinking Clinical Trials

Due to the lack of evidence, indications for red blood cell transfusion in patients with MI are controversial. The MINT trial investigators looked at 30-day mortality and several outcomes including MI, heart failure, stroke, bleeding, infection, and clot. There were 3 primary limitations of the MINT trial.

Trials 130
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Grand Rounds April 14, 2023: RECOVER in Action – Status of Clinical Trial Protocols (Kanecia Zimmerman, PhD, MD, MPH)

Rethinking Clinical Trials

There are many hypothesized causes that may co-exist in the same patient, such as persistent virus or antigens, reactivation of other viruses, uncontrolled immune responses, damage to a wide range of organs and tissues, and injury to blood vessels and abnormal blood clotting.

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CSL doses first patient with haemophilia B gene therapy Hemgenix

Pharmaceutical Technology

The high price tag may be hard to defend , but with lifetime treatment costs for haemophilia B reaching as high as $23m for some, the single-dose treatment could prove far more cost-effective. This protein helps to form blood clots and stop bleeding, with haemophilia B arising when the body is partially or completely unable to produce it.

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Sanofi offers refunds for Cablivi in novel model for rebates

Pharmaceutical Technology

On January 18, Sanofi launched a new warranty program for its rare blood disorder drug Cablivi (caplacizumab), which tackles value-based agreements in a slightly different way. Cablivi stops platelets from clumping together, which prevents small clots, and can be used along with other drugs that suppress the immune system.

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Roctavian okay sets up another gene therapy test for Europe

pharmaphorum

The US biotech claimed conditional marketing approval from the European Commission for Roctavian (valoctocogene roxaparvovec) as a treatment for adults with severe haemophilia A, who don’t have a history of developing the antibodies that inhibit the activity of blood-clotting protein Factor VIII. million spread over five years.

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Hemgenix Approved as First Gene Therapy for Hemophilia B

XTalks

In a pivotal approval, the US Food and Drug Administration (FDA) has given the nod to a new gene therapy called Hemgenix (etranacogene dezaparvovecfor) for the treatment of adults with the genetic blood disorder hemophilia B (congenital Factor IX deficiency). With a list price of $3.5 Hemophilia B primarily affects men.