News

Two potential gene therapies — AMT-060 and AMT-061 (etranacogene dezaparvovec) — lead to safe and long-term increases in factor IX (FIX) activity, as well as fewer bleeds among patients with hemophilia B, according to the therapies’ maker, uniQure. The company announced in a press release that…

The U.S. Food and Drug Administration (FDA) has granted fast track designation to marzeptacog alfa activated (MarzAA), a lab-made version of clotting factor VIIa for the subcutaneous (under-the-skin) treatment of episodic bleeds in hemophilia A and B patients with inhibitors. MarzAA, developed by Catalyst Biosciences, will…

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-060, a first-generation gene therapy candidate by uniQure, continues to safely reduce the number of bleeding episodes and the use of clotting factor IX (FIX) in men with moderate to severe hemophilia B, long-term data from a Phase 1/2 clinical trial show. The therapy also prevented…

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders (NORD) has launched an initiative across the U.S. Its goal is to establish a Rare Disease Advisory Council (RDAC)…

Most people with hemophilia have a positive attitude toward gene therapy, and are willing or “very willing” to receive this treatment, according to a small interview study assessing patient perceptions. These patients reported that they choose their treatments based mainly on five criteria. Specifically, those are…

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a survey conducted by Eurordis-Rare Diseases Europe. “People living with rare diseases in Europe have found themselves caught as collateral…

The first participant was dosed with the investigational therapy marstacimab in a Phase 3 clinical trial — still recruiting eligible patients across the globe — in adolescents and adults with severe hemophilia A or B. Pfizer‘s marstacimab (PF‐06741086) is a human antibody that blocks a…

The healthcare insurance company BCS Financial has partnered with the National Hemophilia Foundation (NHF) to improve care quality and cost for people with hemophilia. BCS Financial provides services primarily for Blue Cross and Blue…

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…