A recent study has revealed that vitamin B3 could offer hope for patients suffering from a rare genetic disorder that causes accelerated aging. As reported by ScienceBlog, researchers found that…
A dedicated Hull resident, Jonny Wightman, is preparing to cycle an impressive 100 miles to raise awareness and funds for rare diseases. As reported by Hull Daily Mail, Jonny’s motivation…
My symptoms began in 2001 and within 6 months I went from functionally uncomfortable to disabled to the point that I could barely lift myself out of bed because more…
Pearl Jam front-man Eddie Vedder takes center stage in the new documentary “Matter of Time,” offering fans and viewers a rare, intimate glimpse into his life as both an artist…
The FDA is taking significant steps to update and streamline its regulatory approach to rare diseases, according to a recent article from BioCentury.com. Recognizing the unique challenges faced by patients,…
Prader-Willi Syndrome (PWS) is a complex, rare genetic disorder known for its hallmark features: insatiable hunger, intellectual and behavioral challenges, and a host of physical health concerns. For families and…
Living with a rare or chronic illness is a journey marked by uncertainty, resilience, and the power of community. For the last 10 years at Patient Worthy, individuals from all…
Alnylam Pharmaceuticals has launched a unique promotional campaign for Givlaari, its treatment for acute hepatic porphyria (AHP), that swaps the usual clinical narratives for something more artistic: dance. As reported…
In a significant stride for global health, the United Nations recently adopted a historic resolution dedicated to rare diseases. This move, as reported by Medics 4 Rare Diseases, celebrated by…
In a groundbreaking medical achievement, physicians have administered the world’s first personalized gene-editing therapy to an infant suffering from a rare and fatal genetic disorder. The innovative treatment, described at…
In the realm of rare diseases, effective communication can be a formidable challenge, especially when symptoms impact speech and expression. Remembering our very first post on May 19, 2015, Patient…
Rebecca Hind, a woman from the UK, endured a life-altering ordeal after what she thought was a simple case of food poisoning turned out to be an extremely rare and…
In the remote town of Serrinha dos Pintos in northeastern Brazil, a rare genetic disorder once shrouded in mystery has shaped the lives of many families for generations. As reported…
Since its inception in 2015, PatientWorthy.com has emerged as a beacon of hope, information, and support for the global rare disease community. From humble beginnings with its very first post,…
An article titled “Accelerated Approval as the New Norm in Gene Therapy for Rare Diseases” from DIA Global Forum digital magazine’s May 2025 issue explores the evolving landscape of regulatory…
Soleno Therapeutics recently announced new findings regarding its investigational drug VYKAT™ XR (diazoxide choline extended-release tablets) for the treatment of Prader-Willi syndrome (PWS), as detailed at GlobeNewswire. The data, which…
Right now, more people than ever are hoping for a kidney transplant — but the system is struggling to keep up. According to Becker’s Clinical Leadership, even a small increase…
According to the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), eating the right diet is essential to managing chronic kidney disease. The NIDDK recommends that patients find…
In honor of Kidney Awareness Month, we would like to congratulate the National Kidney Foundation on celebrating 75 years as an organization fighting against kidney disease and for kidney disease…
Measles cases in Texas and New Mexico have doubled according to Becker's Clinical Leadership surpassing 200. 30 cases have been confirmed in New Mexico, mostly in Lea County which is…
The U.S. Food and Drug Administration (FDA) has approved GSK's combination meningococcal vaccine, Penmenvy, for individuals aged 10 to 25 as reported by Biopharma Dive. This vaccine offers protection against…
Heal Canada is a non-profit dedicated to “patient advocacy, education and collaboration among patient advocacy groups and other healthcare stakeholders.” As part of their extensive offerings of support for patients…
Novo Nordisk plans to seek approval in the US and the EU this year for their drug Mim8 for use on pediatric patients with Hemophilia A after promising results from…
Spinogenix has reported that its Phase II trial of SPG601 for Fragile X Syndrome (FSX) has met its primary goal, according to Clinical Trials Arena. FXS is a genetic disorder…
As reported by Clinical Trials Arena, Genentech and its subsidiary Roche, provided positive Phase III results with the Food and Drug Administration, or the FDA, of their REGENCY trial (NCT04221477)…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
