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…
There are only 11 people in the world diagnosed with Mitochondrial Complex 1 Deficiency. Katherine Faughn, a 7-year-old living in Kentucky is one of these 11. The disease wasn't even…
Today is National Phenylketonuria (PKU) Awareness Day! The United States Senate only very recently voted to designate December 3rd as PKU Awareness Day, in 2012. So let's take a moment…
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…
Caregivers come from all walks of life but they share one common goal- taking care of someone they love who cannot fully take care of themselves. This may mean helping…
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…
"It's funny to say grief as it is not a bereavement but you kind of are grieving the loss of the child you thought you had and the future they…
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…
Stand Up To Cancer (SU2C) has donated a million dollars for a research project dedicated towards a new way of deploying the immune system against pancreatic cancer. This is welcome…
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).…
On November 26, 2018, the FDA approved a new autoinjector for individuals with severe rheumatoid arthritis (RA), giant cell arteritis, and polyarticular or systemic juvenile idiopathic arthritis (aged 2 and older). It's…
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…
This is Part 2 of Alexander's Story, click here to read Part 1. During Alex’s journey Jhoanny had gotten some information from NORD’s website and thought about getting involved. After…
Join us into the future for a moment as we take you through some exciting new developments in health tech! Just last week, the FDA cleared reSET, a mobile app that…
Amy Dahm, who currently lives in Washington, DC, was working as a U.S. diplomat when she was diagnosed with Cushing’s syndrome. After receiving life-saving treatment from the National Institutes of…
The US Food and Drug Administration recently announced the approval of the first biosimilar drug for patients with non-Hodgkin lymphoma in a press release. The drug is known as Truxima…
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