In the shadows of his weight struggles, Sean Mulroney’s journey began at the staggering weight of 687 pounds in 2016. It was a weight that felt like a shackle, chaining…
A major new synthesis of research has confirmed that people living with antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) face a markedly increased risk of developing cancer. Published in Zeitschrift für…
Editor's Note: Patient Worthy is proud to share this article from our friends at Glanzmann's Research Foundation. To see the article in its original form, please click here. Hello! I…
Recent research has brought to light striking genetic differences in chronic lymphocytic leukemia (CLL) among African American (AA) patients, suggesting that their disease may follow a distinct course compared to…
Misfolded proteins found in motor neurons are the cause of muscle weakness and paralysis. People with Amyotrophic Lateral Sclerosis (ALS) generally live two to four years after the onset of…
A recent study published in Clinical Lymphoma, Myeloma, and Leukemia offers new hope for older adults with myelofibrosis (MF), suggesting that allogeneic hematopoietic stem cell transplantation (allo-HCT) remains a viable,…
I’ve spoken regularly with family members and friends about my sons and their diagnosis and about our real-world struggles. I’ve advocated for them at our local pediatrician’s office, educated our…
JCR Pharmaceuticals Co., Ltd., a leader in therapies for rare and genetic diseases, has reached an important milestone in its ongoing collaboration with Alexion, AstraZeneca’s rare disease division. The achievement,…
Jazz Pharmaceuticals plc has taken a major leap in rare cancer treatment with the recent FDA accelerated approval of Modeyso (dordaviprone), the first-ever therapy for relapsed H3 K27M-mutated diffuse midline…
On September 3, 2025, the US Food and Drug Administration (FDA) introduced the Rare Disease Evidence Principles (RDEP) process, a groundbreaking step for drug development targeting ultra-rare genetic diseases. According…
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