More in Hemophilia

FDA Gives Nod to Gene Therapy for Hemophilia A

First gene therapy approved for severe hemophilia A

Jun 29, 2023
FDA APPROVED valoctocogene roxaparvovec-rvox (Roctavian) over a computer rendering of a leaking blood vessel.
'Winners' and 'Losers'; Mandated Sick Leave and Cancer Screening; More AI Advances

News, features, and commentary about cancer-related issues

Mar 03, 2023
Onco Break over a computer rendering of a cancer cell.
FDA Approves Weekly Factor Therapy for Hemophilia A

Factor VIII replacement therapy provides protection from bleeds with once-weekly dosing

Feb 23, 2023
FDA APPROVED Altuviiio over a computer rendering of a clot forming on a damaged blood vessel.
Gene Tx Shows Sustained Bleeding Control in Severe Hemophilia A at 2 Years

Model predicts valoctocogene roxaparvovec could maintain mild hemophilia levels for 5 years

Feb 23, 2023
A computer rendering of a clot forming in a broken blood vessel.
New Class of Factor VIII Replacement Therapy Prevents Bleeds in Severe Hemophilia A

Weekly dosing with efanesoctocog alfa reduced annualized bleeding rate by 77%

Jan 25, 2023
A computer rendering of red blood cells and platelets in a blood vessel.
FDA OKs First Gene Therapy for Hemophilia B

Hemgenix reduced rate of annual bleeds, eliminated need for factor IX prophylaxis therapy

Nov 22, 2022
Hemophilia in blood.
Year in Review: Hemophilia

Gene therapy continues to show promise in clinical trials, but is it ready for the clinic?

Nov 17, 2022
2022 YEAR IN REVIEW HEMOPHILIA over a computer rendering of red blood cells and platelets during hemostasis.
'Unparalleled Times' in Hematology, but Workforce Issues Persist

"This is the most exciting time ever" to be a hematologist, says ASH President Jane Winter, MD

Sep 26, 2022
Lessons From a Lifelong Patient Turned Oncologist

Eric Winer, MD, talks about how growing up with hemophilia helped him connect with patients

Aug 02, 2022
An illustration of a physician holding a patient's hand with the text The Doctor's Art - A podcast on Meaning in Medicine
Sustained Normal Factor IX Levels Seen With Gene Therapy in Hemophilia B

Small study suggests normal levels can be achieved with low vector doses of FLT180a

Jul 26, 2022
A photo of capsules in a petri dish laying a printed graph of DNA sequencing
Gene Therapy Impresses in Severe Hemophilia A

Valoctocogene roxaparvovec significantly increased factor VIII activity, reduced bleeding

Mar 16, 2022
A computer rendering of blood clotting in an epidermis cross section with a puncture
Fitusiran Prophylaxis Cuts Bleeding in Hemophilia A/B

Monthly infusion vs on-demand bypassing agents effective in patients with or without inhibitors

Dec 14, 2021
ASH over a photo of the Georgia World Conference Center in Atlanta, GA.
Hemophilia Gene Therapy Looks Like a Winner

Most patients do well in 3-year phase I/II study

Nov 17, 2021
A computer rendering of red blood cells trapped in a fibrin mesh.
Liver Toxicity in Gene Therapy Trials Could Merit Stricter Entry Criteria

FDA panel weighs in on lower vector doses and role of animal studies

Sep 03, 2021
FDA ADCOMM AAV Gene Therapies over a computer rendering of the liver anatomy.
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