In a story for Drug Development & Delivery, the CEO of the biotechnology company ProQR, Daniel de Boer, recently sat down for an interview. In this interview, Mr. de Boer…
According to a story from dailypost.co.uk, Richard Mason, the millionaire founder of moneysupermarket.com, and and baker Alison Faire's daughters Annabelle (aged 4 years) and Imogen (aged 7 years) don't have…
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Marten Devlieger’s sister Karen, a cystic fibrosis patient, died of the disease at the age of thirty-three. According to a recent article in CTV News Canada, Marten was also diagnosed…
Geisinger was founded by Abigail Geisinger over 100 years ago. It now has 13 hospitals and 2 research centers. They just released a new study examining the severity of symptoms…
According to a story from theage.com.au, a remarkable turn of fate would bring together two women who would have otherwise never known each other---and save both of their lives in…
LETHBRIDGE — A Taber, Alta., man is calling on government to take action and help grant Canadians with cystic fibrosis better access to breakthrough treatments. Marten Devlieger was diagnosed with…
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A recent article in the Washington Post tells the story of a newly-approved cystic fibrosis drug that represents thirty years of scientific work and dedication. The defective gene that causes cystic…
According to a story from scienceblog.com, a recent study has revealed that large portion of cystic fibrosis cases recorded in the Dominican Republic and Puerto Rico are linked to genetic…
According to a story from statnews.com, the approval of the new cystic fibrosis drug Trikafta has caused quite a buzz in this rare disease community. While this new drug offers…
"We went from being able to treat no patients to being able to treat nearly all patients in seven years." 90% of cystic fibrosis (CF) patients now have a new treatment…
According to a story from The Washington Post, data regarding the recently approved drug Trikafta, which is designed to treat cystic fibrosis, was so convincing that the US Food and…
According to a story from BioSpace, the biopharmaceutical company AzuRx BioPharma Inc. recently announced that the first patient has been treated in the company's phase 2 clinical trial. This trial…
According to a story from BioSpace, the US Food and Drug Administration (FDA), has officially approved the drug Trikafta as a treatment for cystic fibrosis, a rare disease that causes…
When Frankie Avila first got his lung transplant, he was told that, like most organs that are transplanted, his lungs only have a certain amount of time before they…
According to a story from Medical Xpress, a recently published French study evaluating the impact of the combination drug lumacaftor-ivacaftor (marketed as Orkambi) concluded that the drug had the potential…
According to a story from abc.net.au, Darren Bullock is getting pretty long in the tooth---at least for a cystic fibrosis patient. He is 44 years old, and his lungs currently…
According to a story from the Cystic Fibrosis Foundation's Community Blog, the internet allows for access to a trove of knowledge and information literally at your fingertips. This has naturally…
According to a story from the Cystic Fibrosis Foundation's Community Blog, the internet allows for access to a trove of knowledge and information literally at your fingertips. This had naturally…
According to a story from The Sunday Post, it is an interesting time to be a cystic fibrosis patient in the UK these days. Just weeks after The Scottish Medicines…
According to a story from The Guardian, Jeremy Corbyn, the leader of the UK Labour Party, announced a remarkable plan at the most recent Labour party conference that has the…
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…
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