Bleeding Disorders
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.
Latest News
AAP guidance helps distinguish bleeding disorders from abuse
Laboratory testing of bleeding cannot always rule out abuse, just as a history of trauma may not rule out a bleeding disorder or other medical...
From the Journals
No more injections after one-off gene therapy in hemophilia B
“Normal factor IX levels can be achieved in patients with severe or moderately severe hemophilia B with the use of relatively low vector doses of...
News
Will the headache field embrace rofecoxib?
The drug formerly known as Vioxx, withdrawn in 2004 over cardiovascular safety concerns, may be given new life as an acute treatment for migraine...
Conference Coverage
Debated: Nonfactor versus gene therapy for hemophilia
Hemophilia A patients have the option of enrolling in clinical trials for gene therapy, but many are satisfied with the efficacy of subcutaneous...
From the Journals
Experts opine on hemophilia treatments
For hemophilia A prophylaxis, debate continues over factor versus non-factor replacement therapy, optimal trough levels, and more.
Feature
Specialists hail new era in hemophilia treatment
New medications are transforming the lives of patients with hemophilia A, and more treatments are on the horizon. Rarer hemophilia B still poses...
From the Journals
‘Time is blood’: Researchers devise shortcut to AHA diagnosis
Researchers call for a potentially life-saving new protocol that can slash delays in confirming a rare hemophilia.