According to a story from Global Knock, nine year old Ashton Leeds has been living an active life again lately, but a year ago, things were not looking up for…
According to a story from pm360online.com, the pharmaceutical company Roche recently announced that its investigational therapy risdiplam has awarded PRIME designation from the European Medicines Agency (EMA). The drug has…
According to a story from Oncology Nurse Advisor, a recent study found that patients with immune thrombocytopenia do not carry the same understanding as their doctors do when it comes…
According to a story from NTD, a former NFL cheerleader successfully diagnosed her own rare disease, pectus excavatum, after her symptoms were dismissed by her doctors as nothing more than…
According to a story from philly.com, Markelle Fultz, who plays as a guard for the Philadelphia 76ers, was recently diagnosed with thoracic outlet syndrome. It has not been good news…
For a long time, we've known that, at least in North America, men develop cancer at higher rates than women. However, there is still some mystery concerning the impact of…
According to a story from Debra of America, a new Phase 1 clinical trial being operated by Stanford University is currently recruiting participants. This trial will be a test of…
According to a story from Cornwall Live, parents Ben Keverne and Hannah Chapman were recently devastated by the loss of their daughter Maisie. The little girl was diagnosed with mitochondrial…
Charcot-Marie-Tooth disease (CMT), is a rare neurological condition. It's hereditary, and it affects the way nerves communicate with muscles. There are different types of CMT, determined by which genes are…
According to a story from pm360online.com, the drug developer Zynerba Pharmaceuticals, Inc., recently announced the release of data from an open label, 12 month study of the company's experimental product…
Angelman syndrome impedes the brain's ability to distinguish background noise from whats important. This is a result of a reduced tonic inhibition, caused by a loss of function of the…
According to a story from liverpoolecho.co.uk, Isabel Morison is a 39-year-old music teacher with a nine year old son Dylan and a six year old daughter, Lucy. Last March, she…
According to a story from Able Magazine, 37 year old Victoria Clutton recently got her first job thanks to the efforts of the UK nonprofit organization ASTRiiD (Available Skills for Training,…
According to a story from USA Today, the day that Stephanie Herfel first picked up her Siberian husky Sierra was easily one of the most important of her life. This…
In August, Duke Cancer Center's Patient Support Program had a backlog of 150 wigs. That means 150 cancer patients who didn't have access to an item that, while simple, provides…
We wrote back in 2015 about Beyond the Diagnosis, a traveling art exhibit featuring portraits of children with rare diseases curated by the Rare Disease United Foundation. Its latest stop was…
Hereditary Angioedemia (HAE) results when the blood is missing the C1-inhibitor protein. It causes unpredictable and sudden attacks of swelling on various parts of the body. It can affect practically…
To be quite frank, Lyme disease is an under-researched condition. If you look at the data concerning how many clinical trials are developed each year for rare conditions, Lyme disease…
One Chinese scientist has completed a research project that has shocked many in the medical community. Two twin baby girls born in November of 2018 where the recipients of his…
Happy Wednesday! With Hanukah wrapped up, and Christmas just around the corner, things are getting pretty crazy. Take a second to relax and check out these four stories. First, we…
According to a story from BioSpace, the cancer drug company Gibson Oncology, LLC has recently announced that the US Food and Drug Administration (FDA) has awarded Rare Pediatric Disease designation…
At Patient Worthy, we are dedicated to the covering the latest stories about rare diseases that appear in the news cycle. While this is an important part of our mission…
According to a story from Proactive Investors, the biotechnology company Conatus Pharmaceuticals recently reported that the company's drug Emricasan failed to achieve the primary endpoint in a Phase 2b clinical…
According to a story from the Greenock Telegraph, twelve year old Megan McGarva, who was born with CDLK5 deficiency, will soon have to face a serious spinal operation. She has…
According to a story from EurekAlert!, the 60th Annual Meeting of the American Society of Hematology featured a number of different presentations of the latest research from St. Jude Children's…
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