It's official: May is Cystic Fibrosis Awareness Month! So myself, and the Patient Worthy team, are excited to bring you some stories to help build your awareness and increase patient…
May is recognized annually as Cystic Fibrosis (CF) Awareness Month. Each year, this rare disease community comes together in order to help spread awareness about cystic fibrosis among the general…
Trevor O'Sullivan Trevor O'Sullivan, who lives in Ireland, has cystic fibrosis (CF). He has undergone two liver transplants. He was the 42nd person in the country to have this surgery.…
By Jodee Redmond from In The Cloud Copy At one time, hearing a cystic fibrosis (CF) diagnosis was tantamount to a death sentence for a young person. The median survival…
When the 31-year-old woman showed up to Massachusetts General Hospital with a nasty respiratory infection, she was somewhat used to it. In fact, she had been plagued with respiratory problems…
U.S. company, Vertex Pharmaceuticals, is the creator of three treatments for cystic fibrosis: Orkambi, Symdeco, und Kalydeco. These drugs were unavailable in Switzerland, as Vertex and Swiss health officials were…
For Mallory Holloway, precautions to avoid lung infections have been a part of her daily life for a long time. This is because she lives with cystic fibrosis, a rare…
According to the Daily Beast, the increasing spread of COVID-19 is causing real problems around the globe - and not just in the way that you might think. This viral…
According to an article in the Baker City Herald, a non-profit organization has launched a website designed to support and educate patients with bronchiectasis. NTM Info & Research advocates for patients…
Arrowhead Pharmaceuticals has recently submitted an application to the New Zealand Medicines and Medical Devices Safety Authority for the clearance of a clinical trial. They want to test their cystic…
If you're worried or anxious because of COVID-19, you're not alone. If wondering about the intersection of rare diseases and COVID-19 only increases that anxiety, well, we're with you…
While many patients are grateful for their doctors and treatments, how many know why they are so passionate about what they do? In an interview with Dr. Patrick Flume,…
According to a release on Public News Service, the state of West Virginia just implemented a new bipartisan law to support individuals with rare diseases and their families, a particular…
According to a story from Fierce Biotech, there have been some impressive advances in the treatment of cystic fibrosis recently. The latest milestone was in developing and approving the drug…
Cystic fibrosis is caused by a mutation in the gene CFTR. This gene involves a protein that affects the chloride on cells; without this chloride the surface of the…
My American Nurse recently carried an uplifting article showing increasingly positive changes for cystic fibrosis (CF) patients. A network of over one hundred thirty centers throughout the U.S. provide…
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…
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…
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