Columnist Jennifer Lynne wraps up a series with hemophilia advocate Lee Hall, who emphasizes the importance of learning our history.

Medically reviewed by Doru Paul, MD Key Takeaways Hemophilia A is a bleeding disorder caused by a deficiency in factor VIII. Treatment options for hemophilia A include replacing factor VIII and gene ...

Findings showed concizumab-treated patients had an 86% reduction in annualized bleeding rate compared with no prophylaxis. The Food and Drug Administration (FDA) has approved Alhemo ® (concizumab-mtci ...

Henry Ford Health has treated the first Michigan patient outside clinical trials with Roctavian, the newly approved gene ...

Less than a year after approval, Pfizer said it plans to discontinue its hemophilia B gene therapy fidanacogene elaparvovec (Beqvez) across all global markets, citing weak demand. "Pfizer has made the ...

Gene therapy is part of an increasingly large collection of research fields: those with a huge, useless backlog of innovations. Gene therapy researchers have spent decades developing amazing, ...

Cell and gene therapies harbor great hopes for biomedicine. They are becoming increasingly important for diseases that are currently difficult or impossible to treat—for example, aggressive forms of ...

In the past three years, gene therapy has reshaped what's possible in hemophilia treatment for patients 18 and older. But a key question remains: How soon will these advances reach children? At the ...

According to multiple media reports on Thursday, Pfizer Inc. (NYSE:PFE) has terminated the global development and commercialization of its hemophilia B gene therapy, Beqvez. In April 2024, the FDA ...

Karnataka launches 'Kusuma Sanjeevini' program, providing free prophylactic treatment and transport for hemophilia patients, enhancing their quality of life.

I didn’t learn about hemophilia from a textbook; I grew up with it in my home. My brother had hemophilia, and our family lived the realities of this rare bleeding disorder every day.