Lots of people with both rare and more prevalent diseases are helped by animals, but the terms service animal, emotional support animal, and therapy animal are often misunderstood, and many…
Garret Schuster was just six months old when he was diagnosed with hemophilia. He went to see a pediatrician to treat an ear infection but the doctor sent him for…
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…
Niemann-Pick Disease Type C is a lysosomal storage disorder caused by malfunction of the NPC1 protein. This protein's dysfunction results in a buildup of lipids in the lysosomes. As a…
For the last 3 decades, the only treatment that has been available for hereditary transthyretin-meditated amyloidosis was a liver transplant. The condition is caused by abnormal transthyretin proteins. These proteins…
According to an article published by MD Magazine, valbenazine, a VMAT2 inhibitor, has proven to be effective in treating patients with Tardive Dyskinesia (TD). Before valbenazine's FDA approval last year,…
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…
According to a story from Buzzfeed News, Stephen Hillenburg, the creator of the smash hit children's cartoon SpongeBob SquarePants has passed away at age 57. Last year, Hillenburg had revealed…
According to a story from MarketWatch, the biopharmaceutical company Rhythm Pharmaceuticals, Inc., recently presented interim data from a Phase 2 trial of setmelanotide as a treatment for obesity associated with…
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 Parkinson's News Today, more and more research is indicating the exercise can have many significant benefits for Parkinson's disease patients. While there are no therapies…
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…
According to a story from finanznachrichten.de, the UK drug development company AstraZeneca recently announced that the US Food and Drug Administration (FDA) has granted Orphan Drug designation for the company's medical…
According to a story from pm360online.com, the drug developer Novartis has recently announced that the company's gene therapy Luxturna has gained approval in the EU as a treatment for Leber's…
The following story includes discussion of suicide. According to a story from EurekAlert!, a recent study has revealed that survivors of head and neck cancer are four times more likely…
Ready for a dose of inspiration to get you through the Monday after a long weekend? We got you covered! Meet Alex Biagi from Colorado - who in 2008 was diagnosed…
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