Note: This story was updated Jan. 8, 2021, to clarify that the second set of experiments in mice and non-human primates used a research construct, not the gene therapy. Generation Bio has announced new data showing that its investigational non-viral gene therapy platform can increase levels of the clotting…
Prime Therapeutics and Takeda will work together to establish the value of Advate, relative to its costs and benefits, as a treatment for hemophilia A. According to Prime, work comparing factors like total health care costs and emergency hospital visits allow the company to better evaluate and distinguish…
Funding has been set for the RESET-HA Phase 3 clinical trial evaluating Tremeau Pharmaceuticals‘ TRM-201 (rofecoxib) in people with hemophilic arthropathy — a painful and degenerative disease caused by recurrent bleeding in the joints. The private investment firm Gurnet Point Capital also will support other aspects of TRM-201‘s…
Dosing has been resumed in ongoing clinical trials in the U.S. testing the investigational medication fitusiran in adults and adolescents with hemophilia, Sanofi announced in a press release. The company had placed a voluntary dosing hold on its full clinical development program for fitusiran at the end of…
A web-based software to help personalize dosing regimens for hemophilia A patients being treated with Adynovate is now available in the U.S., according to Takeda. The software, called myPKFiT, previously was available only for individuals being treated with Advate. For Adynovate, it is to be used by…
AMT-061 (etranacogene dezaparvovec), an investigational gene therapy uniQure is developing to treat hemophilia B, successfully increased factor IX (FIX) activity and controlled bleeding — while markedly reducing the need for other treatments — in the Phase 3 HOPE-B clinical trial, top-line data show. These findings will be presented at…
Due to the identification of new adverse events, Sanofi Genzyme has placed a dosing hold on its full clinical development program for fitusiran, an investigational treatment for hemophilia. The decision was announced in a joint statement from the World Federation of Hemophilia, the European…
Manual therapy, in combination with passive stretching, can reduce the frequency of joint bleeds and improve joint health in people with hemophilia, a small study shows. The study, “The effectiveness of manual therapy in addition to passive stretching exercises in the treatment of patients with haemophilic…
The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases. “The RareLaunch program is central to NORD’s mission and history — community…
Most people with hemophilia who undergo knee replacement surgery have favorable outcomes, including less pain and improved function, a new single-center study from South Korea reports. The study, “Mid-to Long-Term Survival of Total Knee Arthroplasty in Hemophilic Arthropathy,” was published in the Journal of…
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