We all know that changing habits of behavior changes our life experiences, but putting this to practice is where a lot of us get stuck. Either we doubt our ability,…
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…
Editor's Note: Patient Worthy is honored to share this patient story, originally authored by Jeri Burtchell. For the last month or so I’ve been trying to come to grips with…
This week, the FDA approved the first blood-based diagnostic test for Alzheimer's disease (AD). The test, known as "Lumipulse G pTau 217/β-Amyloid 1-42 Plasma Ratio," is designed to detect amyloid…
I have had cervical dystonia since 2001. It was very severe for many years, but over time I have been able to gain significant control over my symptoms using a…
Do you know about Lafora Disease? This rare genetic disease begins during adolescence. Your child starts their life healthy and whole, but then they decline, cognitively and physically, until they…
My name is Elena, mother to Jaicion. In 2021, I was pregnant with Jaicion, and the doctors ran tests that they would run on any mother-to-be. My doctor called me…
Myrovlytis Trust, a UK-based charity and Patient Worthy partner, has developed a new resource for those facing osteosarcoma. This devastating cancer often occurs in childhood, teen, and young adult years.…
My name is Sarah. I am a solo mother to a beautiful five-year-old named Ensley! I’m not quite sure where to begin, but my world abruptly changed post-COVID-infection: January 7th…
In 2023, Marley Mansour of Rowlett, Texas and her family were told that Marley had an extremely rare genetic condition called NARS1 disorder. Now, Marley is the world’s first person…
The results released from the Phase III ESSENCE clinical trial, and recently published by Medical Xpress and in the NEJM, found that the drug semaglutide is effective in the treatment…
Raiden Messerli and his family thought that he had an ordinary case of the flu. Brandi Lewis, Raiden's mother, discussed in an interview with KCCI8 News how she still vividly…
Acknowledgment: This patient story is sponsored by Day One Biopharmaceuticals and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing…
This article is a continuation of a previous story, and is an excerpt from original author Isabella Cristobal's post. To read the first part of the article, please click here.…
Pericarditis is a condition in which the sac that surrounds and protects the heart becomes inflamed. It is most frequently caused by a viral infection and often impacts people in…
Professor Ronald Breslow was a chemist at Columbia University who, in 1958, proposed the theory that vitamin B1 (thiamine) could regulate metabolism pathways in the body by stabilizing a reactive…
Let’s start at the beginning of my long journey. My name is Laura, and around fifteen years ago, I was diagnosed with type-2 diabetes and prescribed medication to control it.…
Immune dysfunction and inflammation have been found to play significant roles in Alzheimer's disease. An analysis of brain tissue in an animal model reveals a potential for immune system dysfunction at…
When asked about the emotions they felt when they were diagnosed, many members of the rare disease community recall a sense of loneliness. Many of us turn to advocacy group…
Current research indicates that children and adolescents who were unvaccinated during the pandemic were much more likely to develop Long COVID. Researchers led by Perelman Medical School at the University of…
This isn’t a cute extra. It’s essential… Let’s stop calling it fluff and start calling it what it is: trauma-informed care in action. Medical play isn’t just a feel-good initiative…
Humans have been using tobacco as a drug for more than 12,000 years. It wasn't until the early-20th century that the link between tobacco and lung cancer (and other health problems)…
From Diagnosis to Determination: My Friedreich Ataxia Journey I was always clumsy. I could never walk in a straight line down the street, bumping into my parents and getting told…
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