In the UK, a think tank determines access to rare disease drugs based on their perception of an individual's quality of life. That idea has also been developed in the…
Amyotrophic lateral sclerosis (ALS) is a condition which affects the bodies nerve cells. It's neurodegenerative and progressive, causing the patient muscle weakness and muscle atrophy. It eventually results in paralysis.…
Mantle cell lymphoma is caused by a genetic mutation which produces cancerous white blood cells. Of course, these are the cells which fight infection in a normal body. Therefore in…
President Herbert Walker Bush passed away on Friday, November 30th at the age of 94. News coverage of his life has reminded us all of the great legacy he leaves…
According to a story from Stanford News, a team of researchers affiliated with Stanford University have successfully developed a new technique for studying high-grade serous ovarian cancer, which is one…
According to a story from BioPortfolio, the drug developer Novartis recently announced that the US Food and Drug Administration (FDA) has granted the company's investigational gene therapy AVXS-101, also known…
According to a story from pm360online.com, the pharmaceutical company Zogenix Inc. recently announced that its experimental drug candidate ZX008, also known as Fintepla®, has performed well in the company's latest…
Behold the power of sheer will, determination, and tenacity! New York teen Hannea Milliman overcame the rare disorder Parsonage Turner Syndrome - which causes severe pain in the shoulder and arm -…
When Deborah, a New Yorker, flew over to Paris to do an internship for an architecture office, she never knew she would be flying across the world and meeting…
According to a story from BioPortfolio, the gene therapy company Rocket Pharmaceuticals has recently announced that the company's investigational gene therapy RP-L102 has earned both Fast Track and Regenerative Medicine…
According to a story from Reuters, the drug developer BioMarin is looking to make a name for itself in the hemophilia community in a very unique way. The company has…
According to a story from statnews.com, the recent FDA approval of a new treatment for Lambert-Eaton myasthenic syndrome could actually be bad news for patients. As it turns out, the…
As Leah approached her late twenties she was, in her own words, a “tank.” She kept up with her ex-Marine husband’s work outs and ran every day. She was the…
As part of the NFL's "My Cause, My Cleats" awareness project, New York Giants Running back Saquon Barkley chose 22q11.2 deletion syndrome (22q) as his cause to highlight! And while that…
The first week of December is Crohn's & Colitis Awareness Week! Courtesy of the Crohn's & Colitis Foundation, there are a few things we can do as rare disease advocates to…
According to a story from Grimsby Live, the family of six year old Melissa Stark, who has Williams syndrome, has transformed their house into a Santa Claus themed wonderland of…
Lambert-Eaton Myasthenic Syndrome (LEMS) is an autoimmune disorder. It's a rare condition characterized by muscle weakness in the limbs. When the muscle weakness includes the respiratory muscles, the disease can…
Tiffany Rowe was 15 when she accomplished her biggest childhood dream. She met Michael Jackson at a live concert and actually got to dance on stage with him. Over…
There's some good news for the hereditary angioedema patient community this week! Shire, a global biotechnology company focused on rare diseases, announced in the Journal of the American Medical Association (JAMA) publication…
According to a story from Medical Xpress, a team of scientists affiliated with the University of Granada have developed a new potential treatment for a form of mitochondrial disease that…
According to a story from Science Alert, an ugly and unpleasant tale involving the devastating rare genetic illness known as Huntington's disease will culminate in a lawsuit. The outcome of…
According to a story from Xconomy, the drug developer Vertex Pharmaceuticals recently announced that a three drug combination treatment for the rare lung disorder cystic fibrosis has performed well in…
According to a story from PR Newswire, the biopharmaceutical company Constant Pharmaceuticals recently announced that it plans to develop its experimental compound TXA127 as a therapy for epidermolysis bullosa (EB).…
At Patient Worthy, we are dedicated to stories about the latest in rare disease news and research, as well as posting stories from rare disease patients themselves. However, there are thousands…
On November 26, 2018, a new drug was given accelerated approval by the FDA. Not only that, but it was granted orphan drug designation and breakthrough therapy designation. The drug…
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