Genetic disorders are caused by genetic mutations (changes) in your gene. Over the past decade especially, researchers increasingly explore genome editing options to fix or address genetic diseases at the…
According to a story from BioPharma Dive, the US Food and Drug Administration (FDA) has recently approved Beqvez, a gene therapy from pharmaceutical giant Pfizer intended to treat hemophilia B.…
According to a story from Reuters Events, hemophilia treatment has seen significant advancements in recent years. However, female patients continue to face difficulties that often lead to disparities in quality…
For the first time ever, people living with severe hemophilia A will have access to a gene therapy solution designed to treat their condition at the source. In a…
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…
Caring is Sharing and Getting Involved. On April 17th Landmarks all over the world will be asked to ‘Light It Red’ in honor of Hemophilia Day. The theme of this…
Approved for use in 2017, emicizumab—marketed under the brand name Hemlibra—has shown immense benefits in the treatment of hemophilia A. But researchers wanted to better understand how emicizumab, used…
Shifts in treatment paradigms can improve the lives of countless patients and contribute to better overall outcomes. In a news release from February 23, 2023, Sanofi and international biopharmaceutical…
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®).…
Their son, Caeleb, was only eleven months old when he developed a severe bleeding episode. The family had just moved from Houston to New Mexico and they had not yet…
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…
In the past, researchers have observed a relationship between hemophilia and bone-related issues. However, while this observation is helpful, there have been few studies examining the prevalence of bone problems…
In 1996, the World Federation of Hemophilia (WFH) launched its Humanitarian Aid Program; it later expanded the program in 2016 to better reach and support patients with hemophilia. The program…
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…
Millions of people worldwide are affected by bleeding disorders such as hemophilia, von Willebrand disease (vWD), Glanzmann thrombasthenia, and more. In 1986, President Ronald Reagan sought to raise awareness around…
In a recent press release, the National Hemophilia Foundation announced it has started a new campaign called “Start the Conversation” which will initiate conversations around inheritable blood disorders. Bleeding Disorders…
According to a story from Al Jazeera, the country of Panama is the only one in Central America with a law on the books that is specifically related to rare…
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.…
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