According to a story from the Sunday Post, sisters Shona and Kirsty Young, aged 24 and 29 years respectively, were both born with the devastating rare disease cystic fibrosis. However,…
According to a story from Market Watch, the biotechnology company Vertex Pharmaceuticals, Inc. has announced recently that the US Food and Drug Administration (FDA) has accepted the company's New Drug…
At the heart of rare disease treatments is research. Currently, 95% of all rare diseases still have no approved therapies. Although there has been an increased focus on rare diseases…
According to a story from Drug Development Technology, the drug developer Translate Bio has recently reported interim results from its phase 1/2 clinical trial. This trial is testing the company's…
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Researchers at the University of Tokyo have discovered that one segment of DNA is responsible for four rare diseases. As reported in Science Daily, the university team also believes that…
According to a story from Raconteur, as many as 3.5 million people living in the UK are expected to affected by some type of rare disease at some point during…
According to a press release from Aridis Pharmaceuticals, the Company has received Orphan Drug designation from the European Medicines Agency for its experimental cystic fibrosis-related lung infection treatment AR-501. The…
Vertex Pharmaceuticals announced the submission of a New Drug Application (NDA) to the FDA for the VX-445 (elexacaftor), tezacaftor and ivacaftor triple combination regimen for treating Cystic Fibrosis (CF). This…
No Cure, No Pay Emile Voest, an oncologist in the Netherlands, conceived the idea of a no cure, no pay model. After witnessing the exorbitant cost of drugs, some of…
Rare diseases can leave patients feeling isolated and suffering in silence across the globe. A lot of work has been done to raise global awareness about common and devastating…
Cleveland Browns wide receiver Jarvis Landry has been affected personally by CF; he lost his high-school sweetheart Mya Zimmer in July of 2015 after her long battle with the rare…
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We often see reports about the latest superbug in the news. Superbugs affect approximately two million people each year and of those, about 23,000 people will not survive. The…
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…
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