Currently, about 90% of cystic fibrosis (CF) patients have a treatment option. This percentage jumped from 50% to 90% upon the approval of Trikafta. However, the remaining 10% of CF…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
Science Direct recently published a Respiratory Medicine Case Report that may impact future Cystic Fibrosis (CF) treatment. The case involves adult brothers herein referred to for purposes of this…
Have you ever heard of newborn screening? Basically, newborn screening is a public health service which can identify potential hormone-related, genetic, or metabolic conditions. Since early identification is so crucial…
Unfortunately, clinical trials do not always go the way that researchers intend. Sometimes, issues arise during early or preclinical studies which cause enough concern to stop or pause the…
Ahead of Cystic Fibrosis Week in the UK, 37-year-old Marc Cotterill, who lives with the disease, has given a video presentation to the European Cystic Fibrosis Conference on the theme…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
In a news release from June 9, 2021, biotechnology company Vertex Pharmaceuticals, Inc. ("Vertex") shared that its therapy TRIKAFTA (elexacaftor/texacaftor/ivacaftor and ivacaftor) was approved for expanded use. Now, the treatment…
Cystic fibrosis patients around the world are fighting to gain access to a range of drugs known as CFTR modulator therapies that could have a hugely beneficial impact on their…
In a study published on Hindawi, a group of patients with severe cystic fibrosis that were awaiting a lung transplant underwent a telerehabilitation program to maintain their lung function. The…
Happy Friday! This week we have a moving patient story about how one mother's pregnancy led to an acromegaly diagnosis. Also, have you heard of Hereditary Tyrosinemia Type 1? If not,…
The month of May is recognized annually as CF (Cystic Fibrosis) Awareness Month, a time meant to help spread awareness about the genetic disorder among the medical community and the…
Each year, researchers make advances in the medical field which assist with the identification, diagnosis, and treatment of patients with rare conditions. According to Genetic Engineering & Biotechnology News, one…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
A recent study published in Cell Reports has uncovered a mechanism where germs living within the lungs of cystic fibrosis (CF) patients produce a type of carbohydrate slime that minimizes the efficacy…
The Cystic Fibrosis Foundation is dedicated to finding a cure for the rare, genetic disorder. They fund more CF research than any other organization, and their latest effort is a…
Note: This guide was originally published on cysticfibrosis.com Written by Imogene The cystic fibrosis community is at high risk for serious complications from COVID-19. Through a recent survey of the…
Translate Bio has been moving forward with the development of their new drug candidate, MRT5005. This mRNA therapy was created to address the underlying cause of cystic fibrosis (CF), and…
Laura Bonnell’s two children have cystic fibrosis (CF). This condition affects the respiratory system and can severely impact patient’s immune system. She explains how she didn’t own a single N95…
Many rare disease patients understand the financial burdens of living with a rare disease. As the therapies are only supporting a small number of patients, the price tag is often…
According to BioMedWire, biopharmaceutical company AzurRx BioPharma ("AzurRx") decided, in mid-November, to advance a series of clinical trials evaluating MS1819 for patients with cystic fibrosis. In particular, the clinical…
Selma Santos has met many nurses over the course of her life. Having a daughter with cystic fibrosis means spending a lot of time in the hospital or at doctor's…
Dan Hopkins is a bowler who has recently raised over $21,000 for cystic fibrosis (CF) in a 65 hour long bowl-a-thon. While he may be exhausted and sore, he is…
On November 27, biotechnology company Vertex Pharmaceuticals Inc. ("Vertex") announced the approval of SYMKEVI with KALYDECO for the treatment of pediatric patients with cystic fibrosis (CF), says GuruFocus. The…
As reported in Warta Saya; for kids with cystic fibrosis (CF), a daily exercise needed to clear the lungs of the disease's characteristic build-up of mucus can feel long and…
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