According to a story from Charcot-Marie-Tooth News, a new study highlights the advances that have been made in the last decade for treating rare diseases that affect children. However, the…
Caption: David Fajgenbaum (right) and I pose for a photo a few years ago in Philadelphia. Credit: National Disease Research Interchange, Philadelphia Tomorrow is Rare Disease Day at NIH, marking…
Janet Woodcock, Director of the FDA’s Center for Drug Evaluation and Research (CDER) recently gave an interview to the publication FDA Voices about the agency’s approval in 2019 of a…
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On February 3, 2020, twenty-seven newly installed flags of the European Union stood in the gleaming lobby of the new European Medicines Agency (EMA) headquarters in Amsterdam. The Charcot-Marie-Tooth…
According to a story from Salon, the Quality Adjusted Life Year (QALY) is a statistical measure that is used in order to calculate the value of a certain medication. First…
The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two…
Proteostasis Therapeutics has just announced results from their ex-vivo study of PTI CFTR modulators using organoids from patients living with cystic fibrosis (CF). These organoids are genetically identical, and have…
According to a story from newsroom.heart.org, one of the most substantial domestic policy achievements of the Obama administration was the passage of the Affordable Care Act (ACA). Since its passage,…
According to a story from the Irish Medical Times, Ireland's Health Service Executive (HSE) has recently announced the extension of a reimbursement agreement between the agency and the pharmaceutical company…
According to a story from the Manchester Evening News, Tom Miller turned 30 years old in January 2019. For Tom, that age was more of a milestone than it was…
Sometimes in moments of tragedy, there is a bright silver lining that helps us learn, and that's exactly what Amber Briggle was aiming to do. Back in October, Amber posted…
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At times there seems to be no happy medium in life. A recent article in Bloomberg claims that the speed at which the FDA is approving newly developed drugs is…
The holiday season and end of the year/decade is upon us! As schedules slow down and we begin to reflect, I too began to take stock of the experiences that…
According to a story from globalnews.ca, Andre and Joshua Laroque are nine and seven years old respectively. The brothers, being close in age, spend a lot of time together and…
Many artists draw inspiration from events in their lives. Dylan Mortimer is no exception, as his art is inspired by his cystic fibrosis. He creates art that mimics lungs,…
According to a story from thejournal.ie, a recent study has discovered a new method for bringing down inflammation in cystic fibrosis. The study authors have concluded that the approach could…
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…
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