Hemophilia News Today

January 17, 2022January 17, 2022

Men Talk About Hemophilia’s Toll, Gaps in Care in Canadian Study

A hemophilia diagnosis takes a toll on the social and mental health of men, who often feel misunderstood and excluded from activities such as sports, a Canadian interview-based study reports. ... Read more

January 14, 2022January 15, 2022

Low-dose ITI Successful in 80% of Hemophilia A Children, Study Shows

Hemophilia News Today

Low-dose immune tolerance induction (ITI) — a type of treatment that can be used to eliminate inhibitors that limit the effectiveness of replacement therapies — achieved partial success in 80% ... Read more

January 12, 2022January 12, 2022

Benefits of Roctavian, Hem A Gene Therapy, Holding for 2 or More Years

Hemophilia News Today

A single dose of the experimental gene therapy Roctavian (valoctocogene roxaparvovec) continues to prevent bleeds and the need for preventive treatment, or prophylaxis, over at least two years in men ... Read more

January 10, 2022January 10, 2022

2seventy Bio, Novo Nordisk Expand Partnership to Develop Gene Editing

Hemophilia News Today

2seventy Bio has expanded its collaboration with Novo Nordisk to develop a gene-editing approach to treat hemophilia A. The new venture adds to an ongoing partnership between 2seventy’s parent company, ... Read more

January 7, 2022January 8, 2022

Trial of Hemophilia B Gene Therapy BBM-H901 Begins Dosing in China

Hemophilia News Today

The first man with hemophilia B has been dosed in a clinical trial in China to evaluate Belief BioMed’s investigational one-time gene therapy BBM-H901. According to the company, BBM-H901 is ... Read more

January 3, 2022January 3, 2022

Scholarship Pays Tribute to the Legacy of Late Patient Jason Fulton

COVID-19 News, Hemophilia News Today

Jason Fulton was accepted to the University of the Pacific in Stockton, California, to pursue a graduate degree in pharmacology and help hemophilia patients like himself. But the 22-year-old knew ... Read more

December 29, 2021December 30, 2021

Webinar on Joint Replacements and Bleeding Disorders Opens HFA Series

Hemophilia News Today

Next year’s Hemophilia Federation of America’s (HFA) monthly educational series for patients, caregivers, and healthcare providers opens on Jan. 18 at 7 p.m. EST with a presentation about joint replacements ... Read more

December 22, 2021December 22, 2021

FLT180a for Hemophilia B Keeps Normal Factor IX Levels for 3+ Years

Hemophilia News Today

In a Phase 1/2 dose-finding study called B-AMAZE, FLT180a gene therapy from Freeline Therapeutics brought about sustained levels of factor IX, the clotting factor that is missing in people with hemophilia ... Read more

December 20, 2021December 20, 2021

Gene Therapy SB-525 Controls Bleedings in Men for at Least 2 Years

Hemophilia News Today

A single dose of the investigational gene therapy SB-525 (giroctocogene fitelparvovec) continues to prevent bleeds and the need for prophylaxis, or preventive treatment, for at least two years in men with ... Read more

December 17, 2021December 17, 2021

EMA Speeds Approval Process for Hemophilia B Gene Therapy

Hemophilia News Today

The European Medicines Agency (EMA) has approved an accelerated assessment request for etranacogene dezaparvovec, an experimental gene therapy for hemophilia B. The decision means that, once an application is submitted ... Read more

December 15, 2021December 15, 2021

Fitusiran Reduced Bleeding Regardless of Inhibitor Status

Hemophilia News Today

Monthly treatment with the investigational medication fitusiran can reduce bleeding in people with hemophilia A and B, with or without inhibitors, according to data from two Phase 3 clinical trials. ... Read more

December 13, 2021December 13, 2021

Collision Risk in Active Hemophilia A Patients Tied to Greater Bleed Risk

Hemophilia News Today

Physical activities with a high risk of a collision are linked with a greater risk of bleeding in people with moderately severe hemophilia A, a six-month U.S. study reports. This risk ... Read more

December 10, 2021December 11, 2021

HOPE-B Trial Update: Gene Therapy Candidate AMT-061 Meeting Goals

Hemophilia News Today

After 18 months, the investigational gene therapy AMT-061 (etranacogene dezaparvovec) continued to prevent bleeds in men with moderate-to-severe hemophilia B, according to top-line data from the Phase 3 HOPE-B clinical ... Read more

December 8, 2021December 8, 2021

BioMatrix Announces Winners of Its 2021 Memorial Scholarship Program

Hemophilia News Today

BioMatrix Specialty Pharmacy has announced this year’s winners of its memorial scholarship program, designed to help people with hemophilia and other bleeding disorders pursue higher education. Six scholarships, each worth ... Read more