Written When Diagnosed On January 15th, 2024 my life changed forever. I felt my right breast turn completely solid. I knew something wasn’t right, so I went to my doctor.…
A recent article from Pharmaceutical Manufacturer highlights a significant achievement in the rare disease sector, as Rare Disease Biotech announced reaching a major milestone in the development of its new…
I had learned that the many twists and turns on our rare disease path were similar to the families we met Originally published on May 11, 2022, this article explores celebrates author…
In October 2021, I was diagnosed with a rare, possibly life-threatening autoimmune disease called Myasthenia gravis. Some people said I had to “reinvent myself,” because indeed I could not, and…
A recent article published in the Orphanet Journal of Rare Diseases highlights the launch and goals of RARE-Impact, a new European project designed to enhance the diagnosis, care, and overall…
Sixty-five-year-old JoEllen Zembruski-Ruple had been a cancer patient at the revered Sloan Kettering center in January of this year. Her treatment for squamous cell carcinoma began with three chemotherapy pills.…
For Part 1 of this series provided to you by TREND Communities, please click here. On July 10, 2024, TREND Community released an impactful new report titled “Prader-Willi Syndrome: Perspectives…
In an inspiring act of love and advocacy, Joburg resident Nthabiseng Mokoena has completed a remarkable 35-day walk from Johannesburg to Cape Town to raise awareness for a rare disease…
Editor’s Note: This is the second part of an ongoing story. The third and final part will be published Tuesday, June 25th. To read part one, click here. The initial…
Researchers have discovered a potential “window” whereby babies born with inherited diseases may be treated with gene therapy sent directly into their circulatory systems. The window consists of circulating stem…
Launched in May 2015, Patient Worthy set out with a bold mission: to amplify the voices of rare disease and chronic illness patients, and to connect and inform a community…
This Father’s Day, Patient Worthy honors the strength, resilience, and unwavering dedication of fathers of children with rare diseases. Their journeys inspire us all and remind us of the vital…
HepaRegeniX GmbH has announced the dosing of the first patient in its Phase Ib clinical trial of HRX-215, a novel small-molecule inhibitor designed to promote liver regeneration. This marks a…
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…
In 2019 I presented with dark purple spots on my legs, and my neurologist decided to do lab work. My labs showed a high IGM level, so I was then…
Disclaimer: The views and opinions expressed in this article are those of the author and do not necessarily reflect the official policy or position of Patient Worthy. At just 5…
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…
The FDA recently informed Muscular Dystrophy News that it has granted orphan drug designation to the experimental drug ISX9-CPC, a product of the IPS Heart company located in Houston, Texas.…
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…
In 2014 the U.S. experienced a nationwide outbreak of EV-D68, raising concerns about children’s respiratory health. Acute flaccid myelitis disease (AFM) also spiked. EV-D68 is defined as a group of…
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,…
Editor's Note: This is the first part of an ongoing story. The second part will be published Tuesday, June 17th. The fluorescent lights of the hospital room hummed, a stark…
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…
Researchers at the UK Dementia Institute in London (UK DRI) have identified a common denominator for various gene mutations that cause amyotrophic lateral sclerosis (ALS). A new CRISPR study shed…
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