
How A Rare Genetic Condition May Be Treated by A Simple Vitamin
A recent article from SciTechDaily describes the promising approach instituted at a private, non-profit biomedical research firm in San Francisco that incorporates data science, AI,

A recent article from SciTechDaily describes the promising approach instituted at a private, non-profit biomedical research firm in San Francisco that incorporates data science, AI,

According to a recent press release, Artios Pharma has begun dosing patients in a global Phase 2 clinical trial evaluating its investigational DNA polymerase theta

A significant advancement in antiviral therapy has emerged with the successful development of a CRISPR-Cas13d-based system designed to combat hepatitis E virus (HEV) infections. This

In a recent press release from Pfizer, it was shared that the European Commission (EC) has broadened the approved use of Pfizer’s marstacimab (brand name

A recent report by the Manila Times highlighted Madrigal Pharmaceuticals’ unveiling of a series of new analyses and real-world findings supporting the therapeutic profile of

UniQure has announced plans to submit its groundbreaking Huntington’s disease gene therapy, AMT-130, for regulatory approval in the United Kingdom later this year, marking a

According to a recent report on MedTechDive, Guardant Health has received U.S. Food and Drug Administration (FDA) approval for an updated version of its liquid

Editor’s Note: Patient Worthy is honored to present this story by Heather Doyle, originally published by the United Porphyrias Association. To see the article in

Editor’s Note: This piece, originally written by Kayla DeSonier, was shared with us by our friends at Elephants & Tea. To see the article in

Editor’s Note: This article was shared with us by our friends at the Cure NF2 Foundation, in honor of May 22nd being World NF2 Awareness

Exelixis has taken a major step forward in its colorectal cancer development pipeline by securing supplies of Merck’s Keytruda for an upcoming phase 3 clinical

Betty Vertin is a mother, writer and a fierce defender of the decisions she and her husband Jason made about the seven children they brought

According to the CDC, a look back at April 2026 cumulative reports showed more than 139 pediatric deaths with approximately 250,000 people hospitalized. In simple

Atossa Therapeutics Inc. has achieved a significant regulatory milestone with the U.S. Food and Drug Administration’s grant of Rare Pediatric Disease (RPD) designation to (Z)-endoxifen

An article from University of Michigan highlighted Jolanta Grembecka and her husband Tomasz Cierpicki, both assistant professors in U of M’s Department of Pathology, who

Milenia Makani is an abstract artist living in London. She lives with Hypermobile Ehlers-Danlos Syndrome (hEDS), and has launched an international art fundraiser to support

What happens when a pharmaceutical first-mover discovers its historic achievement may not be a blockbuster? Arvinas and Pfizer found an answer this week by handing

What is Parkinson’s Disease – known as PD for short? Parkinson’s Disease was first discovered in 1817 by a British doctor named James Parkinson. From
Multifocal Motor Neuropathy (MMN) is a rare neurological condition that primarily affects movement rather than sensation. Because symptoms often begin gradually and may resemble other neuromuscular disorders, understanding the signs can help support earlier recognition and diagnosis. Awareness matters—especially for conditions that many people have never heard of until they or someone they love is affected.
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For many people living with rare diseases, changes in the voice can be more than just hoarseness. Neurological, muscular, and other underlying conditions can affect the ability to speak clearly, communicate comfortably, and be understood by others. Raising awareness of symptoms like these helps highlight the often-overlooked ways rare diseases impact daily life and why every patient deserves to have their voice heard.
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Living with a rare or chronic illness means even “taking it easy” isn’t always easy. Some days, rest helps. Other days, your body has completely different plans.
To everyone navigating flare-ups, fatigue, pain, and the unpredictability that people don’t always see—we see you. 💙
#ChronicIllness #RareDisease #InvisibleIllness #ChronicIllnessWarrior #RareDiseaseAwareness #PatientWorthy
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