In a recent press release from Pfizer, it was shared that the European Commission (EC) has broadened the approved use of Pfizer’s marstacimab (brand name HYMPAVZI), extending its indication to…
The biotechnology sector witnessed a striking demonstration of expedited drug development this week, with Boehringer Ingelheim receiving FDA approval for an innovative lung cancer therapy in less than two months,…
Pfizer has announced breakthrough Phase 3 results for HYMPAVZI™ (marstacimab), a once-weekly subcutaneous therapy that could redefine care for people with hemophilia A or B who have developed inhibitors, as…
Gene therapy for hemophilia B has reached a significant milestone: more than a decade after a single treatment, patients continue to experience stable benefits with minimal safety concerns, according to…
A recent study highlighted in Hematology Advisor explores the challenges of monitoring patients with hemophilia A after gene therapy, specifically focusing on the reliability of laboratory assays used to measure…
The treatment landscape for hemophilia B has taken a historic leap forward, as the first patient in the UK’s National Health Service (NHS) has received Hemgenix, a pioneering gene therapy…
Novo Nordisk plans to seek approval in the US and the EU this year for their drug Mim8 for use on pediatric patients with Hemophilia A after promising results from…
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.…
When young Emmett Cordes initially complained of knee pain, his mother attributed it to his active nature. However, as the pain persisted, a visit to the doctor and subsequent…
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…
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…
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 families,…
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…
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