CS ECHO: Salima’s Fight Against Cowden Syndrome
Cowden Syndrome. A name that sounds almost gentle, until you realize it carries generations of uncertainty. It’s caused by changes in the PTEN gene and can affect the skin, thyroid,…
Continue Reading
CS ECHO: Salima’s Fight Against Cowden Syndrome
Record-Breaking Pig Kidney Transplant Marks Milestone in Xenotransplantation Research
In a groundbreaking moment for transplant medicine, Massachusetts General Hospital (MGH) has concluded its second successful xenotransplantation of a genetically modified pig kidney into a human patient. Tim Andrews, a…
Continue Reading
Record-Breaking Pig Kidney Transplant Marks Milestone in Xenotransplantation Research
FDA Accelerates PORT-77 for Rare Sunlight-Sensitive Disorders
Portal Therapeutics, in partnership with GondolaBio, has achieved a milestone with the FDA granting orphan drug and fast track status to its oral therapy candidate, PORT-77. This investigational drug, according…
Continue Reading
FDA Accelerates PORT-77 for Rare Sunlight-Sensitive Disorders
Worms Lead the Way in Rare Disease Drug Discovery
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…
Continue Reading
Worms Lead the Way in Rare Disease Drug Discovery
From Scars to Strength: My Journey Toward Visibility and Healing
Sometimes our greatest lessons come from the moments that change us forever. Healing has not happened all at once for me. It has been a lifelong journey that began with…
Continue Reading
From Scars to Strength: My Journey Toward Visibility and Healing
Unlocking a New Era: Illumina’s 5-Base Solution Bridges Genomics and Epigenomics for Multiomic Discovery
Illumina, Inc. has unveiled its groundbreaking 5-base solution, setting a new standard for multiomic research by enabling scientists to simultaneously analyze genomic and epigenomic information from a single sample. According…
Continue Reading
Unlocking a New Era: Illumina’s 5-Base Solution Bridges Genomics and Epigenomics for Multiomic Discovery
New Lancet Study Highlights Severe Malnutrition in Gaza
A recently published study in The Lancet reports that over 54,600 children living in Gaza are severely malnourished. In addition, as of August 2025, there are few therapeutic options available…
Continue Reading
New Lancet Study Highlights Severe Malnutrition in Gaza
Merck Advances Tulisokibart’s Clinical Reach: New Phase 2b Trials Target Three Immune Diseases
Merck has announced a significant expansion of its clinical development program for tulisokibart (MK-7240), an investigational monoclonal antibody targeting the inflammatory mediator TL1A. Reported by Drugs.com, in October 2025, the…
Continue Reading
Merck Advances Tulisokibart’s Clinical Reach: New Phase 2b Trials Target Three Immune Diseases
Seasonal Affective Disorder and What We Can Do About It
If you live in the Northern Hemisphere, Fall has quickly passed and Winter is upon us. The beginning of Fall is one of favorite times of the year with the…
Continue Reading
Seasonal Affective Disorder and What We Can Do About It
Navigating Medical Complexity Through Shared Decision-Making
Editor's Note: Patient Worthy is proud to share this from our friends at the Courageous Parents Network. Throughout our lives we are faced with decisions of many kinds. Some involve…
Continue Reading
Navigating Medical Complexity Through Shared Decision-Making
Genetic Risk for Dilated Cardiomyopathy May Elevate Vulnerability to Secondary Cardiomyopathies
Emerging research highlights the role of genetic predisposition in the development of secondary cardiomyopathies triggered by environmental stressors such as pregnancy, alcohol use, and cancer therapy. Findings published in JAMA…
Continue Reading
Genetic Risk for Dilated Cardiomyopathy May Elevate Vulnerability to Secondary Cardiomyopathies
The Weight of a Decision
Editor's Note: We're proud to be the first to share this story by Blair Young Whitworth, a Parent Champion of the Courageous Parents Network. There were so many anxious years…
Continue Reading
The Weight of a Decision
Brewing Good Health: New Research Reveals Coffee’s Powerful Gut Benefits
Coffee lovers have long celebrated their favorite beverage for its taste and energy boost, but recent research offers another compelling reason to savor that daily cup: coffee directly and substantially…
Continue Reading
Brewing Good Health: New Research Reveals Coffee’s Powerful Gut Benefits
Beyond the Parachute: Why Patient Recruitment Partners Belong in the Cockpit
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”…
Continue Reading
Beyond the Parachute: Why Patient Recruitment Partners Belong in the Cockpit
Redefining Autoimmune Care: T Cell Engagers and the Promise of Immune Reset
Autoimmune diseases affect an estimated 400 to 600 million people globally, posing a growing challenge for healthcare systems and patients alike. Traditionally, treatment has focused on symptom management through broad…
Continue Reading
Redefining Autoimmune Care: T Cell Engagers and the Promise of Immune Reset
FDA Decisions Could Reshape Pharma Landscape in Late 2025
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.…
Continue Reading
FDA Decisions Could Reshape Pharma Landscape in Late 2025
One-in-a-Trillion: A Texas Family’s Journey Through Rare Disease, Loss, and Unshakable Faith
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…
Continue Reading
One-in-a-Trillion: A Texas Family’s Journey Through Rare Disease, Loss, and Unshakable Faith
How Safe Is Tylenol Really? The Answer Is Not As Simple As You May Think
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…
Continue Reading
How Safe Is Tylenol Really? The Answer Is Not As Simple As You May Think
Alera Bio Takes Bold Step Toward First Treatment for MCT-8 Deficiency with AB-101 Program
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…
Continue Reading
Alera Bio Takes Bold Step Toward First Treatment for MCT-8 Deficiency with AB-101 Program
In-Home Homicides of Young Children In their Own Home More than Doubled from 2001 to 2020
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…
Continue Reading
In-Home Homicides of Young Children In their Own Home More than Doubled from 2001 to 2020
Rare Cancer Explorer 1.0 (RaCE 1.0): A Game-Changer for Integrative Rare Cancer Research
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…
Continue Reading
Rare Cancer Explorer 1.0 (RaCE 1.0): A Game-Changer for Integrative Rare Cancer Research
JCR Pharmaceuticals and Alexion Mark Key Progress in Neurodegenerative Disease Collaboration
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,…
Continue Reading
JCR Pharmaceuticals and Alexion Mark Key Progress in Neurodegenerative Disease Collaboration
FDA’s RDEP Framework: Paving the Way for New Treatments in Ultra-Rare Genetic Diseases
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…
Continue Reading
FDA’s RDEP Framework: Paving the Way for New Treatments in Ultra-Rare Genetic Diseases
What Is the Blood-Brain Barrier? (Part 2)
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…
Continue Reading
What Is the Blood-Brain Barrier? (Part 2)
- Go to the previous page
- 1
- …
- 4
- 5
- 6
- 7
- 8
- 9
- 10
- …
- 303
- Go to the next page
