Editor's Note: Patient Worthy is proud to bring you this story-share, originally written by Gwen Pike, founder of the podcast Empowerment in Illness. When I was in my twenties, life was…
A new study published in Aging-US reveals that Parkinson’s disease (PD) patients diagnosed in their 80s are significantly more likely to exhibit amyloid-beta buildup, which is an early marker of…
In a significant advancement for rare disease treatment in Japan, the Ministry of Health, Labour and Welfare (MHLW) has approved Bylvay® (odevixibat) for the management of pruritus associated with progressive…
When a woman hears the words, “You have cancer,” her world shifts in an instant. The journey ahead—marked by treatment, uncertainty, and emotional upheaval—can feel isolating, even for the strongest…
Each year, Patient Worthy covers the American College of Medical Genetics conference, the leading source of both genetic education for health care professionals and a leading source of new information…
As 2025 winds down, the pharmaceutical industry is bracing for a series of pivotal FDA decisions that could redefine treatment paradigms and reshape competitive dynamics across several major therapeutic areas.…
In a landmark step for personalized cancer care, the National Comprehensive Cancer Network® (NCCN®) has launched the NCCN Guidelines Navigator™—a cutting-edge digital platform that redefines how clinicians access and use…
Editor's Note: Patient Worthy is honored to bring you this patient story-share from Michael Grivas. For more information and resources, please visit the SADS Foundation. My name is Michael Grivas,…
In a quiet corner of Texas, Jenna Hornbuckle is living a story so medically rare, doctors estimate its probability in the trillionth range. A single mother of two, Jenna is…
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…
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