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…
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…
According to an article published by CheckOrphan, LEO Pharma and PellePharm have announced a new multi-million dollar partnership together with the aim of advancing research for rare skin diseases that…
The Asia-Pacific Economic Cooperation (APEC) has 21 members including the United States, China, Canada, Australia, Peru, Russia, and Thailand to name a few. They work to encourage the interdependence of the…
The world is recognizing more every day just how important rare disease research is. Yes, rare diseases are rare but they affect millions of people worldwide. The more we can…
According to a story from tgen.org, a team of scientists from the Translational Genomics Research Institute (TGEN) have recently initiated a study that will test the effectiveness of liquid biopsy…
According to a story from Health Canal, a team of researchers led by Dr. Mohammad Siddiqui of Virginia Commonwealth University (VCU) recently released a study which further highlights the relationship…
Last week the Biotechnology Innovation Organization released a letter to the FDA, commenting on the latter's draft of a document that proposes a new set of guidelines for the treatment…
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