According to a story from The Charlotte Observer, Chris Younger was 18 when he first was diagnosed with X-linked hypophosphatemia, a rare disease that affects the bones. However, he had…
A recent article in ANCA Vasculitis News highlighted a Chinese study of ANCA-associated vasculitis (AAV), a group of autoimmune diseases. An earlier Chinese study suggested that the risk of…
A recent study has found that undergoing chemotherapy after having surgery for a rare kidney cancer reduces the chance of cancer relapse by 50%. The recent study was published…
By Danielle Bradshaw from In the Cloud Copy. Many of us probably don’t think too hard about our medical records on a day to day basis; which is fair -…
By Danielle Bradshaw from In the Cloud Copy. Right before Thanksgiving last year, Sami Winters, an East Bay resident, was admitted to Benioff Children’s Hospital in Oakland, California. It’s now…
By Jodee Redmond from In The Cloud Copy The results of a case study have found that dantrolene can be used to treat involuntary muscle contractions (dystonia) in children with…
As originally reported in Biospace, Cushing disease, a disease due to excessive cortisol, has recently had its first ever treatment option approved by the FDA for adult patients who are…
Hey, hand-washing friends! We hope everyone is staying safe and clean right now. Of course, the world is reckoning with Covid-19 right now-- and the rare space is no except.…
According to a story from businesswire.com, the Bristol Myers Squibb Company recently released the topline results from its phase 3 clinical trial. This study was testing a combination treatment consisting…
According to a story from Healing Hugs Haven, the rapid spread of COVID-19 around the world has been getting a lot of airtime in the news lately, and for good…
As originally reported in Targeted Oncology, a man was diagnosed with EGFR-Mutated Non-Small Cell Lung Cancer, also known as EGFR-NSCLC. The 60 year old had a previous medical history that…
By Danielle Bradshaw from In The Cloud Copy Pompe disease is the lack of a specific protein within the body that helps break down glycogen - a complex sugar that…
As originally reported in the Cincinnati Children's, Brittney writes how she came into the world of rare diseases when her and her husband Michael decided to adopt a baby who…
As reported in Creaky Joints and Healing Hugs Haven; the media has been overtaken by coronavirus on the move. Rare disease patients need to know what that means and what…
Rare Disease Institute The Rare Disease Institute (RDI) is a new initiative aimed at improving collaboration between rare disease stakeholders. The aim is to facilitate faster diagnosis and better care…
NPR News recently featured an article announcing that scientists at Portland’s Casey Eye Institute have attempted to use CRISPR to edit a gene with the DNA still in place.…
According to a recent article in Healio, the National Institute of Health states that one-third of strokes occurring for the first time to people under fifty can be linked to…
1 in 10 people suffer from a rare disease, but when I talk about writing for Patient Worthy, it’s usually the first time people have had a conversation or really…
According to an article in BioPortfolio, Magenta Therapeutics, a biotechnology company based in Cambridge, Mass., recently announced updates to its Phase II trial of MGTA-456 for the treatment of…
This year, the entire Patient Worthy team traveled up to Washington, DC for Rare Disease Week 2020. This event is always a big one for the rare disease patient community.…
Susan Mann was seven years old when she was diagnosed as having Hashimoto’s thyroiditis, an autoimmune disease that affects the thyroid. According to a recent article in the online…
As originally reported in Biospace, two research networks dedicated to forming bridges between rare disease research around the world will share their niches to promote discovery for rare diseases. TriNetX…
Todd Mercer, a fifty-two year old Michigan resident and father of two, recently called into STAT’S Podcast The Readout LOUD to discuss the difficulty he has had getting enrolled…
Hope everybody is catching up after Rare Disease Week! We're so glad we got to see advocates and patients at RDW making the rare voice heard in DC last week.…
Why are rare diseases so difficult to study? Is it just their rarity? Or is it also industry challenges? Unfortunately, industry has a lot to do with it. Clinical Trial…
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