Roctavian, Hem A Gene Therapy, Sustains Drop in Bleeding Rates, Phase 3 Data Show
Roctavian, an investigational gene therapy, significantly reduced bleeding rates and the need for other treatments in men with…
Articles by Marisa Wexler, MS
Non-viral Gene Therapy Shows Promise in Hemophilia A Animal Models
Note: This story was updated Jan. 8, 2021, to clarify that the second set of experiments in mice and non-human…
Cost-Benefit Analysis Set for Advate, Hemophilia A Treatment
Prime Therapeutics and Takeda will work together to establish the value of Advate, relative to its costs and…
Funding Set for TRM-201 Trial for Joint Disease in Hemophilia
Funding has been set for the RESET-HA Phase 3 clinical trial evaluating Tremeau Pharmaceuticals‘ TRM-201 (rofecoxib) in people with…
Dosing of Fitusiran in U.S. Hemophilia Trials to Resume
Dosing has been resumed in ongoing clinical trials in the U.S. testing the investigational medication fitusiran in adults and adolescents…
myPKFiT Software for Adynovate Now Available in the US
A web-based software to help personalize dosing regimens for hemophilia A patients being treated with Adynovate is now…
AMT-061 Increases FIX Activity, Reduces Bleeding in Phase 3 Trial, Data Show
AMT-061 (etranacogene dezaparvovec), an investigational gene therapy uniQure is developing to treat hemophilia B, successfully increased factor IX (FIX)…
Sanofi Genzyme Places Trials for Fitusiran on Hold
Due to the identification of new adverse events, Sanofi Genzyme has placed a dosing hold on its full clinical…
Manual Therapy With Passive Stretching Helps Hemophilia Patients’ Joints, Study Finds
Manual therapy, in combination with passive stretching, can reduce the frequency of joint bleeds and improve joint health in people…
NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits
The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops…