Developing novel therapeutics for rare diseases is increasingly important. Understanding the unmet needs of rare disease communities and providing effective, accessible therapies can significantly improve the lives of those affected.…
Anxiety is the apprehension, uncertainty, and fear one feels when anticipating a threatening event or situation, whether the threat is real or imagined. It is often accompanied by restlessness, problems…
A majority of rare diseases are genetic in nature, with an estimated 50-75% of these conditions manifesting in childhood. The Rare Pediatric Disease designation was established to encourage and streamline…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
When it comes to rare disease, rare isn't actually that rare. More than 10,000 rare diseases have been identified. Rare diseases affect more than 350 million people across the globe…
February 15th is recognized annually as International Angelman Day, a day dedicated around the world to spreading awareness among the general public and in the medical field about Angelman syndrome,…
Editor's Note: Chronic conditions and rare diseases don't discriminate, Patient Worthy and its partners are interested in amplifying the voices of those from all identities and backgrounds. If you have…
In late January 2024, the U.S. Food and Drug Administration approved a label expansion for Dupixent (dupilumab). Dupixent, a monoclonal antibody targeting IL-4 and IL-13, is currently used to treat…
Quinnlyn Walters is a bright, bubbly, and active 8-year-old. She loves reading, riding horses, swimming and softball, karate, Star Wars, and spending time outside with her friends and family. But…
On Monday, February 12 the Food and Drug Administration approved Eohilia, a budesonide corticosteroid oral suspension for patients aged 11 years and older with eosinophilic esophagitis (EoE) patients in the…
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
At four and a half years old, Tucker Lewis is thriving. He loves playing with his friends, riding his bike outside, and watching the Atlanta Braves (He's even going to…
Prilenia Therapeutics, Waltham, Massachusetts and Naarden, Netherlands is a biotechnology company with a focus on developing therapeutics to treat neurodevelopmental disorders and neurodegenerative diseases. Prilenia presented data this month at…
According to a story from the Baltimore Sun, a childhood home of Henrietta Lacks - a Black woman often dubbed "the mother of modern medicine" - was unknowingly demolished just…
Biomarkers are objective measures that scientists, researchers, and doctors can use to understand what is going on in your body. For example, your blood pressure can tell doctors about your…
Treatments and management plans exist for chronic obstructive pulmonary disease (COPD), heart failure, and interstitial lung disease (ILD). Yet despite these treatments, many people with these conditions still experience chronic…
We are excited to announce that Patient Worthy's award-winning podcast "Wait, How Do You Spell That? A Rare Disease Podcast" is back with a new episode. This week, Colby is…
Written by Maria Picone, TREND Community Ten years ago, my daughter was born with a rare, complex disease known as Prader-Willi syndrome (PWS). PWS is best known for causing insatiable…
19-year-old Emily Duke was having an amazing time at Auburn University, where she studied hard and spent her spare time as acting Vice President of Alpha Omicron Pi sorority. She…
Metabolic dysfunction-associated steatohepatitis (MASH) unfortunately has no treatments to reverse the disease or its damage. MASH can be managed through weight loss, regular exercise, and blood sugar management. But identifying…
Have you ever heard of Marfan syndrome? This rare inherited disorder affects connective tissue in the body. Connective tissue supports organs and bodily structures like your skeleton, blood vessels, eyes,…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Despite novel treatment advances, hepatocellular carcinoma (HCC) remains difficult to treat. This aggressive cancer is often not found until later stages and not all people with HCC can utilize available…
The primary outcome from the Phase III NETTER-2 clinical trial as reported recently in Globe Newswire, was heralded as extending progression free survival from 8.5 months to 22.8. The participants…
Patient Worthy is excited to highlight Probably Genetic's free genetic testing program for alpha-mannosidosis. The intricate web of rare diseases often leaves individuals and their families searching for answers and…
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