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 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…
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…
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…
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…
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…
Editor's Note: Patient Worthy is proud to share this article from the TESS Research Foundation. To see the article in its original form, please click here. TL;DR — the blood-brain…
Editor's Note: Patient Worthy is proud to share this article with permission from the TESS Research Foundation. To see the article in its original form, 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…
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…
Medetoidine is an animal sedative increasingly being found in human opioid deaths according to published reports in the journal “The Conversation” and shared on MedicalXpress. Synthetic opioids claim thousands of lives…
Capsida Biotherapeutics has suspended a clinical trial of its experimental gene therapy after the first child to receive the treatment died, sending shockwaves through the rare disease and gene therapy…
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…
Stanford University has recently secured a prestigious grant from the U.S. National Institutes of Health (NIH) to accelerate the clinical development of Tiakis Biotech’s novel therapy, Elafin (also called Tiprelestat),…
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…
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…
Ireland has unveiled a new national strategy aimed at transforming the diagnosis and care of the estimated 300,000 people in the country living with rare diseases. According to The Irish…
After cancer treatment, many patients are left with a haunting uncertainty: Is it really gone—and will it stay that way? Even when scans appear clear, questions linger. Now, a newer…
A groundbreaking study from the Weizmann Institute of Science and Sheba Medical Center has brought renewed hope for children with kaposiform lymphangiomatosis (KLA), a rare and life-threatening genetic disorder of…
August 2025 was a landmark month for rare disease innovation, as the U.S. Food and Drug Administration (FDA) granted four historic approvals, each representing a first for their respective conditions,…
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