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…
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…
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…
For many families, when their child is born with developmental delays, there may be weeks, months, or even years that go by without a diagnosis. During this time, parents…
According to a story from accesswire.com, the drug developer BioVie, Inc., recently announced that the US Food and Drug Administration (FDA) has granted Orphan Drug designation to the company's investigational…
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…
Nonalcoholic fatty liver disease (NAFLD) is exactly what it sounds like. It's caused by a buildup of fat cells in the liver, resulting in many complications caused by the thickening…
According to a story from Digital Journal, the biopharmaceutical company Acceleron Pharma, Inc., recently announced that its investigational drug candidate ACE-083 was granted Fast Track designation from the US Food…
Lynch Syndrome Lynch syndrome is a genetic condition which makes an individual more susceptible to certain types of cancers. Specifically, it has been thought to increase risk for developing colorectal…
According to a story from BioPortfolio, Celgene Corporation and bluebird bio, Inc., have recently announced that enrollment for a pivotal Phase 2 study testing an experimental therapy for relapsed/refractory multiple…
There’s an episode of “The Office” that begins when Michael Scott, the branch manager, arrives at work wearing a fake mustache, an attempt to disguise what he believes to be…
Jhoanny’s son Alexander Matthew was born perfectly healthy in 2008. When Alexander was three, Jhoanny noticed he seemed to be drinking a lot of water and sweating a lot. Since…
Stephen Hillenburg, the creator of Nickelodeon's iconic cartoon series “SpongeBob SquarePants,” died Monday of this week after battling amyotrophic lateral sclerosis (or ALS, also known as Lou Gehrig’s disease). What is ALS?…
According to a story from globenewswire.com, the biopharmaceutical company PhaseBio Pharmaceuticals has recently announced that the first patient has been dosed in a Phase 2b clinical trial of the company's…
Just this week, the Food and Drug Administration approved a drug that targets a wide range of cancers based on DNA, rather than where the tumor is in the body. This is being…
According to a story from The Star Online, the government of Malaysia has allotted RM50 million of its 2019 budget for the treatment of numerous rare diseases, stunted growth, hepatitis…
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