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…
"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…
Cystic Fibrosis Cystic Fibrosis (CF) is a rare disease caused by a defected CFTR gene. It results in a buildup of mucus which impairs breathing and makes the lungs susceptible…
The book Five Feet Apart was first released to the public in November 2018 and on March 15th, 2019, a film based on the novel was released into theaters. The…
Rare diseases such as Tourette's syndrome and cystic fibrosis can be, among all of the other challenges that come with their diagnosis, extremely isolating. A recent article on Psychology Today explains…
According to a story from nbcnewyork.com, scientific research on cells was always a challenging task. Trying to study a certain trait used to mean looking at a tissue sample, which…
Each year, Rare Disease Day (February 28th) incites individuals and organizations to investigate and reform their rare disease efforts. This year, the EU Commission has made drastic changes which should…
Rare disease isn't as rare as it sounds. Ten percent of Americans have a rare disease-- and there are almost 7,000 known rare diseases. A rare disease, also known as…
Cystic Fibrosis Cystic fibrosis (CF) is a rare condition that causes mucus to build up in the lungs and the digestive system. It results in chronic lung infections, lung inflammation, and…
Cystic Fibrosis Cystic fibrosis (CF) is a rare disease caused by the lack (or malfunctioning) of the cystic fibrosis transmembrane conductance regulator (CTFR) protein. This occurs when children inherit a mutated…
The Problem One of the greatest challenges in the investigation of nanoscale treatments for people with rare diseases is figuring out how to administer the therapy to the correct part…
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