A Rare Disease Gets Its First FDA-Approved Medicine—And It Works Differently Than Anything Available Before Imagine your immune system getting the signals completely wrong. Instead of protecting you from infection,…
Editor's Note: Patient Worthy is honored to share this article from our friends at Heal Canada, written by Karen Hawthorne. Anemia is one of the most common and serious complications…
Acknowledgment: Patient Worthy is honored to share this story, provided to us by our friends at The AAMDS International Foundation. To see the article in its original format, please click…
In a week of significant developments for the biopharma industry, an FDA advisory committee issued pivotal recommendations on two high-profile blood disorder drugs, while PureTech Health announced a leadership change…
A growing focus on patient-reported outcomes (PROs) is transforming the management of Paroxysmal Nocturnal Hemoglobinuria (PNH) and aplastic anemia, two rare and serious blood disorders. According to a recent article…
People living with blood disorders like sickle cell disease or thalassemia will now have access to a new blood test that will reduce transfusion side effects. England’s National Health Service…
Give the gift of life this holiday season and consider donating your bone marrow to BeTheMatch. Each year, over 18,000 people are diagnosed with a life-threatening blood condition that could…
Anemia is a common symptom for chronic kidney disease (CKD), but there hasn't been much research done on the association between the two or the adverse outcomes which ensue as…
Prior to February 2022, there were no approved treatments for those with cold agglutinin disease (CAD). However, that changed on February 4th, 2022, when the FDA announced that it had…
Just 50 years ago, receiving a diagnosis of severe aplastic anemia could be devastating. While therapeutic options have greatly improved over the past five decades, some doctors have still found…
As reported in Biospace; a Phase 2 study by Bristol-Myers Squibb and Acceleron Pharma Inc. earned itself recognition as among the top six abstracts received by the Presidential Symposium of…
The seventy-seven-year-old patient, Mr. S., had lived with chest discomfort for twenty years. Since bypass surgery or stents were not an option, his cardiologist managed to ease his discomfort through…
Tanner McLeod has been searching for a stem cell donor for a long time. He was diagnosed with sideroblastic anemia at just six months old and has required a transplant…
In 2019, Japan approved the use of EVRENZO (roxadustat) for patients with anemia of chronic kidney disease (CKD) who were also on dialysis. However, the orally administered therapy was not…
In a recent press release, biopharmaceutical company Akebia Therapeutics ("Akebia") announced the publication of the Phase 3 INNO2VATE study design and methodology. The publication examines the INNO2VATE trial, which evaluated…
Austin Jackson is a star football player at the University of Southern California. His entire life consists of football, whether that is training for it, watching it, or playing it.…
Some of us here may have heard of Camp Sunshine -- which provides retreats "combining respite, recreation and support, while enabling hope and promoting joy, for children with life-threatening illnesses…
At four years old, Norah Gratz-Lazarus of California is a sweet, bubbly girl, full of spirit. She's a big sister, and loves jokes, ice cream, and chocolate chip cookies. However, Norah…
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