According to a story from prnewswire.com, the biopharmaceutical company Aridis Pharmaceuticals, Inc. has recently announced that the US Food and Drug Administration (FDA) has awarded Orphan Drug designation to the…
Tannaz Motevalli Tannaz Motevalli is the Exhibition Coordinator at the National Library of Medicine, an artist, a researcher, and a writer. She also lives with cystic fibrosis (CF). Experiencing fewer…
It is 2019, and minority populations still account for fewer than 10% of clinical trial participants. Two decades ago, the "NIH Policy and Guidelines on the Inclusion of Women and…
About a month ago, we reported on a story about an Irish family that is facing deportation from Australia. The family includes Anthony and Christine Hyde along with their three…
According to a story from BBC, 35 year old Kimberly Chard knows that she probably doesn't have much longer to live. Kimberly was born with cystic fibrosis, a rare disorder…
The Problem Orkambi is a drug for cystic fibrosis (CF) created by Vertex. It is effective for approximately 40% of all CF patients. It was licensed in 2015 by the…
The Beginnings Anne Pariser worked for 16 years at the FDA. For part of that time, she was a team leader for the Center for Drug Evaluation and Research where…
Gunnar Esiason, a 28-year-old cystic fibrosis (CF) patient, just delivered an inspirational pre-commencement speech to the 2019 St. Louis University School of Medicine graduating class. Quite frankly, it contains wisdom…
The Cure Rare Disease Foundation The Cure Rare Disease Foundation's mission is to increase collaborative efforts between researchers in order to accelerate the development of customized therapies for individuals living with…
May is Cystic Fibrosis (CF) awareness month! CF is one of the more devastating rare diseases in the community, often affecting children and young people. So let's be extra mindful…
Phage Therapy Phages, or bacteriophages, are the natural enemy of viruses. They are the most abundant organisms in the biosphere. Researchers have used phages as treatments before, however after the…
According to a story from ArcaMax, history was recently made when a 15 year old teen with a severe "superbug" case of Mycobacterium abscessus infection was successfully treating using specially modified…
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A recent article in CBC News describes the heartbreak and struggle of a Canadian couple appealing to the Canadian government to fund medication for their younger son. Both their children, Andre…
According to a story from The Irish Times, a couple's application for permanent residency in Australia has been rejected twice now following an appeal. Anthony and Christine Hyde, along with…
According to a story from express.co.uk, Elle Morris was only 11 years old when she died from a severe lung infection. Elle was born with cystic fibrosis, a serious genetic…
According to a story from EurekAlert!, a group of researchers affiliated with Penn Medicine and Children's Hospital of Philadelphia recently conducted an experiment in which the scientists used CRISPR gene…
According to a story from the Daily Record, cystic fibrosis patient Kelli Gallacher found that she was able to stabilize her disease using the drug Symkevi. Now that her own…
According to a story from Medical Xpress, a recent study conducted by researchers from the UNC School of Medicine has illustrated that the accumulation of sticky, abnormal mucus, along with…
According to a story from Cystic Fibrosis News Today, Elizabeth Rogers, a cystic fibrosis patient, is just one of the many who have been disappointed by the lack of impact…
My first admission is that I actually know quite a bit about ABC's The Bachelor. Yes, that includes The Bachelorette too. I first started watching “ironically“ with my girlfriend, who…
According to a publication from EurekAlert, a recent study suggests a common, typically symptomless form of the herpes virus called cytomegalovirus may cause faster disease progression in cystic fibrosis (CF)…
According to an article at HealthEuropa, including patients and their families as an important component of cystic fibrosis research is a requirement when developing future treatments for the disease. That's…
"The simple idea of a single gene leading to a single disease is more likely to be an exception than a rule." Finding the Disease-Causing Genes Things would be, not…
Zachary and Imogen Jones are brother and sister with cystic fibrosis (CF). While Imogen is older, she received her CF diagnosis after her brother. It was actually through educational programs…
The FDA previously announced their plan to hire 50 new employees specifically to manage the influx of gene therapy applications. Currently, they have approximately 800 applications awaiting review and they…
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