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…
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…
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…
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…
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…
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…
California Rare Disease Advisory Council Virtual Coalition Meeting February 17, 2021 Legislation to create a California Rare Disease Advisory Council (RDAC) will be introduced by State Senator Susan Eggman in…
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…
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…
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