CFTR gene mutations cause cystic fibrosis, a rare genetic condition which causes progressive digestive and respiratory system damage. Yet it can be extremely difficult to treat for those with “stop”…
In a news release from January 4, 2022, biopharmaceutical company SpliSense shared that their drug candidate SPL84-23-1 for cystic fibrosis (CF) earned Orphan Drug designation in both the United States…
In June 2021, Health Canada approved TRIKAFTA (elexacaftor/tezacaftor/ivacaftor) for patients with cystic fibrosis (CF), with at least one F508del mutation, aged 12+. According to a news release from late…
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…
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…
Online Support Group for Cystic Fibrosis Caregivers May 18, 2021 CFRI provides a monthly CF Caregivers Online Support Group, which is open to participants nationwide. The group is facilitated by…
Online Support Group for Adults with Cystic Fibrosis May 17, 2021 CFRI provides a monthly Online Support Group for Adults with CF, which is open to participants nationwide. The group…
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…
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…
Online Support Group for Cystic Fibrosis Caregivers April 20, 2021 CFRI provides a monthly CF Caregivers Online Support Group, which is open to participants nationwide. The group is facilitated by…
Online Support Group for Adults with Cystic Fibrosis April 19, 2021 CFRI provides a monthly Online Support Group for Adults with CF, which is open to participants nationwide. The group…
Virtual CF Winter-to-Spring Mini Retreat March 19-21, 2021 This event will take place fully online and will feature daily exercises, arts and crafts, support group sessions, and education presentations from…
Online Support Group for Cystic Fibrosis Caregivers March 16, 2021 CFRI provides a monthly CF Caregivers Online Support Group, which is open to participants nationwide. The group is facilitated by…
Online Support Group for Adults with Cystic Fibrosis March 15, 2021 CFRI provides a monthly Online Support Group for Adults with CF, which is open to participants nationwide. The group…
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…
Online Support Group for Adults with Cystic Fibrosis February 15, 2021 CFRI provides a monthly Online Support Group for Adults with CF, which is open to participants nationwide. The group…
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…
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