A groundbreaking study from the MRC London Institute of Medical Sciences (LMS) is reshaping how researchers approach treatment development for rare genetic disorders. Led by Dr. André Brown and the…
Editor's Note: Patient Worthy is proud to share this story from GACI Global, originally published by the Italian Osservatorio Malattie Rare (OMaR). ARHR2 – Autosomal Recessive Hypophosphatemic Rickets Type…
Clinical trials often begin with optimism, sites are launched, projections look promising, and timelines are set. But when enrollment stalls, sponsors frequently call-in patient recruitment partners as a last-minute “rescue”…
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…
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…
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…
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,…
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…
Luke McCarthy of Killybegs is preparing to take on the Galway Bay Marathon, not just as a personal fitness goal, but as a heartfelt tribute to his father, Joe, who…
Editor's Note: Patient Worthy is proud to share this article from our friends at the TESS Research Foundation. To see the article in its original format, please click here. TL;DR:…
On Sunday, more than 100 medical experts from across the globe gathered in Rochester for the Mayo Clinic’s four-day “Undiagnosed Hackathon”—a pioneering event dedicated to solving rare diseases that have…
After years of determined advocacy and scientific perseverance, Stealth BioTherapeutics has achieved a landmark victory with the FDA’s accelerated approval of elamipretide (now branded as Forzinity) the first-ever treatment for…
Avacopan, a novel oral therapy for antineutrophil cytoplasmic antibody-associated vasculitis (ANCA-AAV), has changed treatment paradigms for this rare but serious disease. However, until now, a validated technique for measuring avacopan…
In our third installment from our series of reports provided by our partner TREND Community, we are bringing you several reports that analyze the effects of sleep disorders. This week…
An estimated 47% of infants have some form of plagiocephaly (a flat spot on the head), and up to 10% will eventually need a corrective helmet to help reshape their skull. Some…
In a breakthrough study, scientists have uncovered a surprising ally in the fight against cancer: the bacteria living within tumors. According to World Pharma News, an international team led by…
Editor's Note: Patient Worthy is proud to share this story from our friends at No Stomach for Cancer. To see the article in its original form, please click here. In…
Life has a way of catching us off guard when we least expect it. For me, it all started with severe stomach pains that led to an unexpected hospital visit.…
Research into rare diseases has long faced obstacles, from limited patient numbers to the challenge of replicating disease biology in the lab. A new wave of innovation, highlighted in the…
The U.S. Food and Drug Administration (FDA) has introduced a new regulatory framework aimed at improving and clarifying the approval process for drugs targeting rare diseases, also known as orphan…
Editor's Note: Patient Worthy is honored to share this article from our friends at Courageous Parents Network. Parents of well children regularly receive anticipatory guidance–proactive communication about what’s coming in…
Editor's Note: This article is an opinion piece shared with Patient Worthy from our partner network. Opinion pieces may not reflect the views of Patient Worthy, its employees, or any…
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