“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…
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…
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…
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…
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…
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,…
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…
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…
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.…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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.…
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,…
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.…
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 –…
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 –…
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…
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…