The 17th Annual Congress of the European Association for Haemophilia and Allied Disorders took place this year from February 6-9, 2024. During the Congress, stakeholders came together to discuss how…
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…
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…
Our blood has different clotting factors, or protein, that helps clot and stop bleeding after injuries. But when you don't have enough of these proteins, the blood can't clot…
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…
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 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,…
Jo Traunter was told that she was "a bleeder" throughout her life, whether it was by family, doctors, or others. She earned this title after living through decades of heavy…
According to a recent article, researchers reported their findings from a new study that looked at the bleeding patterns and characteristics in infants and toddlers with von Willebrand disease (VWD).…
Receiving a rare disease diagnosis can be difficult, frustrating, and scary. Laura Hamilton, the host of A Place in the Sun, is no stranger to this phenomenon; last year she…
As many patients in the rare disease community know, achieving a diagnosis can be a lengthy and difficult journey. Following this journey, it can still take a while to receive…
Monica and Josh Poynter have recently adopted a nine-year-old boy, Trey, from China. They felt a connection to the boy as he has hemophilia type A, the same rare bleeding…
What are the best treatments and diagnostic approaches for blood disorders like aplastic anemia and myelodysplastic syndromes (MDS)? The UT Southwestern Medical Center is trying to streamline these options and…
Last Tuesday, the FDA rejected BioMarin Pharmaceutical's gene therapy, Roctavian, for patients with hemophilia A. According to ABC News, the gene therapy infusion was meant to be a single treatment…
Many people use art, music, or writing to cope with events in their life. Personally, my platform is poetry; I love the way that I can use words to…
This year, thanks to COVID-19, the European Hematology Association (EHA) took their annual conference online - but that didn't prevent some intriguing new insights and ideas. In a press release,…
Camp Notaclotamongus In Eureka, Missouri, Camp Notaclotamongus has some typical activities for a summer camp. They state that camper goals aim to build friendships, confidence, and personal growth with outdoor…
Big news for those with hemophilia! On Thursday, the FDA approved Hemlibra (manufactured by Genentech), a weekly self-injection for hemophilia A patients who have developed resistance to standard medicines for preventing…
I first became aware of the bleeding disorder hemophilia when I was in high school reading about Nicholas II, the last Russian tsar. His son had severe hemophilia B, which…
When you have a bleeding disorder, you have to be careful doing everyday tasks. It changes your normal routine. It changes your life. For Malaika, her diagnosis of hypoprothrombinemia took…
Immune thrombocytopenia purpura, or ITP, is a bleeding disorder caused by a lower than normal number of platelets, otherwise known as thrombocytes. Platelets are made in the bone marrow and when…
In the world of bleeding disorders, Factor VII deficiency could be considered the “runt of the litter.” And in the “dog eat dog world” of chasing research dollars, it’s a…
Julia Smith was diagnosed with Glanzmann thrombasthenia (GT) at the tender young age of six months through a series of fortunate events. First, her mother Helen, noticed her infant bruised very…
When most people think of bleeding disorders, they tend to think of Hemophilia. And if they know a little more than the average guy or gal—and let’s face it, if…
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