“Being Okay” – Kylie’s NF1 Journey
When we are faced with an obstacle, big or small, we supposedly pick one of two options: to fight it or to run away. Nowadays, they say there are more…
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“Being Okay” – Kylie’s NF1 Journey
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…
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New Lancet Study Highlights Severe Malnutrition in Gaza
Navigating the Challenges of Biomarker Testing in Oncology: Innovations for Low-Yield Samples
Biomarker analysis plays a pivotal role in cancer care and research, guiding the development of targeted therapies and enabling personalized treatment plans. While next-generation sequencing (NGS) has revolutionized the ability…
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Navigating the Challenges of Biomarker Testing in Oncology: Innovations for Low-Yield Samples
Alkermes’ Alixorexton Shows Promise in Narcolepsy Type 1: Vibrance-1 Phase 2 Results to Be Unveiled at World Sleep 2025
Alkermes is set to present detailed positive results from its Vibrance-1 Phase 2 study of alixorexton, a novel, once-daily oral orexin 2 receptor agonist, in patients with narcolepsy type 1…
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Alkermes’ Alixorexton Shows Promise in Narcolepsy Type 1: Vibrance-1 Phase 2 Results to Be Unveiled at World Sleep 2025
11 Foods for a Stronger Liver: What to Eat and Why It Matters
Your liver is a powerhouse organ, responsible for processing nutrients, detoxifying your blood, and managing everything from cholesterol to vitamins. Diet plays a vital role in keeping this organ healthy,…
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11 Foods for a Stronger Liver: What to Eat and Why It Matters
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…
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Merck Advances Tulisokibart’s Clinical Reach: New Phase 2b Trials Target Three Immune Diseases
Why Your Brain Might Be Hijacking Your Wakefulness: Understanding Hypersomnia and Narcolepsy
Excessive daytime sleepiness (EDS) is more than just feeling tired—it can be a debilitating symptom of two distinct neurological disorders: hypersomnia and narcolepsy. While both conditions share the hallmark of…
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Why Your Brain Might Be Hijacking Your Wakefulness: Understanding Hypersomnia and Narcolepsy
Stealth Bio’s Forzinity: FDA Approves First-Ever Treatment for Barth Syndrome
After years of setbacks and uncertainty, Stealth BioTherapeutics has achieved a historic breakthrough with FDA approval of Forzinity (elamipretide), the first treatment for Barth syndrome, an ultrarare, life-limiting genetic disorder.…
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Stealth Bio’s Forzinity: FDA Approves First-Ever Treatment for Barth Syndrome
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…
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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…
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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…
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Genetic Risk for Dilated Cardiomyopathy May Elevate Vulnerability to Secondary Cardiomyopathies
“Downton Abbey” Creator Undergoes Transformative Treatment for Tremors
Lord Julian Fellowes, the Oscar-winning creator of Downton Abbey, has undergone a transformative treatment for essential tremor (ET) using Insightec’s magnetic resonance-guided focused ultrasound (MRgFUS) technology. After living with the…
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“Downton Abbey” Creator Undergoes Transformative Treatment for Tremors
Urgent Message from the American Liver Foundation
Over 9,300 individuals living in the United States are currently waiting for a liver transplant. There is a serious need for liver donors. This message was recently sent out by…
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Urgent Message from the American Liver Foundation
Narcolepsy Unveiled: Understanding Symptoms, Diagnosis, and Evolving Treatments
Narcolepsy is a chronic neurological disorder that disrupts the brain’s ability to regulate the sleep-wake cycle, causing a profound impact on daily life. Characterized by excessive daytime sleepiness, sudden episodes…
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Narcolepsy Unveiled: Understanding Symptoms, Diagnosis, and Evolving Treatments
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…
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The Weight of a Decision
Sanofi’s Gene Therapy for Wet AMD Earns FDA Fast Track Designation
The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to SAR402663, Sanofi’s investigational gene therapy for neovascular age-related macular degeneration (AMD). As seen in a report, this…
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Sanofi’s Gene Therapy for Wet AMD Earns FDA Fast Track Designation
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…
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Brewing Good Health: New Research Reveals Coffee’s Powerful Gut Benefits
Rady’s Lupus Journey: Carried by Faith, Fueled by Hope
I came to the United States from the Philippines in June 2016, full of excitement and dreams. I was ready to build a new life, work hard, and create something…
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Rady’s Lupus Journey: Carried by Faith, Fueled by Hope
Experimental Gene Therapy Slows Huntington’s Disease Progression in Landmark Trial
An experimental gene therapy known as AMT-130 has become the first treatment to successfully slow the progression of Huntington’s disease, marking a potential breakthrough in the fight against neurodegenerative disorders.…
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Experimental Gene Therapy Slows Huntington’s Disease Progression in Landmark Trial
FDA Signals Flexibility as Lexeo’s Gene Therapy for Rare Disease Advances Toward Faster Approval
Lexeo Therapeutics announced a significant regulatory development this week: the U.S. Food and Drug Administration (FDA) is open to a faster approval pathway for the company’s promising gene therapy, LX2006,…
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FDA Signals Flexibility as Lexeo’s Gene Therapy for Rare Disease Advances Toward Faster Approval
Sherry Crutchfield’s Journey with Glanzmann Thrombasthenia
Editor's Note: Patient Worthy is proud to share this story from our friends at the Glanzmann's Research Foundation (GRF). To see the article in its original format, please click here.…
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Sherry Crutchfield’s Journey with Glanzmann Thrombasthenia
MASH (formerly known as NASH) Patient Stories Needed
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 –…
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MASH (formerly known as NASH) Patient Stories Needed
MASH (formerly known as NASH) Patient Stories Needed
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 –…
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MASH (formerly known as NASH) Patient Stories Needed
Unlocking the Future: Sleep Disorder Biomarkers Predict Parkinson’s and Dementia
In a groundbreaking international effort led by Dr. Shady Rahayel of Université de Montréal and reported by MSN.com, researchers have discovered new ways to predict which individuals with isolated REM…
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Unlocking the Future: Sleep Disorder Biomarkers Predict Parkinson’s and Dementia
Datroway Shows Survival Benefit in Late-Stage Trial for Triple-Negative Breast Cancer
AstraZeneca and Daiichi Sankyo have announced promising Phase 3 trial results for Datroway, their investigational antibody-drug conjugate (ADC) targeting triple-negative breast cancer (TNBC). The trial demonstrated that Datroway significantly slowed…
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Datroway Shows Survival Benefit in Late-Stage Trial for Triple-Negative Breast Cancer
