Neoadjuvant therapy, which involves administering treatment before surgery to shrink tumors, has become a cornerstone in breast cancer management. Yet, despite its promise, current clinical decision-making often relies on a…
Pancreatic cancer took Alex Trebek, Michael Landon, Ruth Bader Ginsburg, Luciano Pavarotti, Steve Jobs, Patrick Swayze, and my dear friend Amy. Glioblastoma (GBM) took Senators John McCain and Ted Kennedy,…
Editor's Note: Patient Worthy is honored to share this article from our friends at the Fatty Liver Foundation. We get a lot of questions about Tylenol and pain relievers in…
Your story has the power to educate, motivate, and make a real difference in the lives of others. We are looking for patients with Metabolic Dysfunction-Associated Steatohepatitis, or MASH -…
NS Pharma, Inc., a subsidiary of Nippon Shinyaku Co., Ltd., has received a significant regulatory boost for its experimental treatment targeting Duchenne muscular dystrophy (DMD). As reported in The AI…
Immuneering’s midstage pancreatic deep cyclic MEK protein inhibitor, (DCI), almost doubled the standard overall survival. It is a deep cycle inhibitor that targets key pathways in a pulsed manner. Results…
Editor's Note: Patient Worthy is honored to share this story from our friends at Elephants & Tea. To see the article in its original form, please click here. Original author…
A new analysis has uncovered stark differences in the rates of breast anaplastic large cell lymphoma (ALCL), a rare cancer often linked to breast implants, among women of different racial…
A recent preprint reported by medRxiv explores the complex relationship between genetic mutations and clinical presentation in Colombian patients with congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency (21OHD). While…
Editor's Note: This article was translated into English from Spanish; there may be inconsistencies or minor errors. Sports and the fight against Dravet syndrome will once again go hand in…
Alera Bio, a Chicago-based biotech company dedicated to rare neurological diseases, has announced a significant breakthrough in the quest to treat MCT-8 deficiency, otherwise known as Allan-Herndon-Dudley Syndrome. On September…
A recent study by UCLA researchers of in-home murders of children twelve and under revealed that according to the National Violent Death Reporting System and other agencies, nearly one quarter…
Editor's Note: This opinion piece was originally written by Kim Higbee. The opinions expressed in this article may not be a reflection of Patient Worthy, its employees, or any of…
When Ashley Rowland’s daughter, Aubrie, was diagnosed with a rare mitochondrial disease called AARS2-related leukoencephalopathy before her first birthday, Ashley’s world changed forever. Seven years later, she continues her fight—not…
Rare cancers, defined by their low incidence, collectively constitute nearly a quarter of all cancer diagnoses in the US and Europe, and represent a significant public health challenge. Despite their…
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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