An announcement by Pfizer was recently published in Biospace outlining the results of BASIS its Phase 3 clinical trial (NCT03938792). The drug being studied, marstacimab, met its primary endpoints…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
According to a story from Globe Newswire, the gene therapy company uniQure recently announced that it has received conditional marketing authorization for its gene therapy etranacogene dezaparvovec (marketed as HEMGENIX®).…
The American Hematology Society (ASH) held its 64th Annual Meeting in December 2022. During the Meeting, a variety of stakeholders—including physicians, researchers, and industry members—gathered to discuss research, trends, and…
Currently, the standard-of-care for individuals living with hemophilia B is intravenous factor IX infusions (prophylaxis). These infusions can be time-consuming, require adherence to a strict schedule, and may not…
In 2002, three-year-old Collin Johnson underwent a tonsillectomy. Just a couple of days later, after Collin woke up from a nap, Stormy noticed a large pool of blood on the…
Sometimes, it can be difficult to incentivize researchers and drug developers to focus on solutions for those with rare conditions. The FDA created the Orphan Drug Act to overcome…
According to a recent article in the British publication Latest Page News, doctors at the University College in London and Royal Free London believe that a ‘cure’ is possible within…
Recently, the International Society on Thrombosis and Haemostasis (ISTH) held its 2022 Congress from July 9th to 13th. During the conference, researchers and other stakeholders discussed research, care, and…
The first patient has been dosed in the Phase 1/2 B-LIEVE trial of FLT180a, an investigational treatment for hemophilia B. Freeline Therapeutics, the drug developer, announced the news in a…
In a recent article for Hemophilia News Today, Jennifer Lynne, who is diagnosed with both von Willebrand disease and hemophilia B, stresses the importance of remembering women can have hemophilia…
According to an article from the National Hemophilia Foundation, CSL Behring and uniQure have recently provided an update on their phase III HOPE-B trial. This study is evaluating etranacogene dezaparvovec,…
According to Hemophilia News Today, GENV-HEM, an investigational treatment for patients with hemophilia A and B either with or without clotting factor inhibitors, earned Orphan Drug designation from the FDA.…
In a news release from May 18, 2021, biopharmaceutical company Catalyst Biosciences, Inc. ("Catalyst") shared that the first patient was dosed in the Phase 1/2 MAA-202 clinical trial. During…
According to a story from the National Hemophilia Foundation, the drug company uniQure has provided an update in regards to a case of hepatocellular carcinoma (HCC) that emerged in a…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Mononine, a treatment for hemophilia B, hit the market nearly three decades ago in 1992. Since then, there have been amazing advancements in the knowledge and treatment for this disease.…
By Lauren Thayer from In The Cloud Copy Hemophilia is a medical condition in which a person’s blood clotting ability is severely reduced. This inability to properly clot leads to…
According to Hemophilia News Today, data collected from a Phase 2b trial highlights that the subcutaneously administered DalcA is effective for the treatment of hemophilia B. Find the full press…
According to a story from Hemophilia News Today, the drug developer uniQure has completed its goals for patient enrollment ahead of time for is phase 3 clinical trial. This clinical…
According to a story from MD Edge, a phase 2 clinical trial testing the drug concizumab as a treatment for hemophilia A and hemophilia B demonstrated substantial capability in reducing…
The 2019 Annual Symposium The Coalition for Hemophilia B's annual symposium is the largest annual gathering specifically for hemophilia B patients and their families. Learn the latest in research and…
According to a story from PR Newswire, the genomics medical company Sangamo Therapeutics, Inc., recently announced that the first patient had been dosed in its Phase 1/2 clinical trial which…
According to a story from hemophilia.org, the drug developer uniQure recently announced that the first patient had been dosed in the company's Phase IIb clinical trial. This trial is testing…
According to BioPortfolio, the company UniQure has recently announced that the first hemophilia B patient has been enrolled in their Phase III study of the treatment AMT-061, which is an…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
