Bleeding Disorders
From the Journals
Red-cell donor’s sex does not affect transfusion survival
New study showed that the sex of a red blood cell donor has no bearing on the survival of the transfusion recipient.
From the Journals
Hemophilia A gene therapy under FDA review
Recently published clinical data showed that a costly new gene therapy for hemophilia A was found largely durable at 2 years.
From the Journals
Efanesoctocog alfa treatment: ‘Victory’ over hemophilia A
In a phase 3 study of patients with severe hemophilia A, a new factor VIII injection given weekly prevented almost all bleeding episodes.
News
Europe approves first gene therapy for hemophilia B
Exorbitantly priced gene therapy to treat hemophilia B has been approved for sale across the European Union and European Economic Area.
News
FDA approves once-weekly hemophilia A product
Newly approved by the FDA, this replacement therapy for a rare bleeding disorder has been found to reduce both dosing frequency and bleeds.
From the Journals
Abnormal bleeding common among youth with joint hypermobility
Study suggests that children with generalized joint hypermobility should be routinely screened for abnormal bleeding symptoms and referred as...
Conference Coverage
ITP: Biologic beat placebo, but few patients improved
Exorbitant efgartigimod meets primary endpoint in only 21.8% of patients with chronic disease.
Conference Coverage
Beta-thalassemia: Benefits of gene therapy outweigh costs
Following treatment with betibeglogene autotemcel, some patients went without transfusions for 8 years and counting.
News
FDA approves first gene therapy for hemophilia B
The gene therapy will cost $3.5 million, making it the most expensive treatment to date.
Feature
Post Roe, pregnant SCD patients facing “dire” risks
Doctors warn that lost abortion rights put many pregnant patients with sickle cell disease (SCD) in danger.
Feature
Royal family affliction or not, porphyria is treatable
This rare blood disorder is often misdiagnosed, but a pricey new drug offers relief.