In 2020, the U.S. FDA granted Orphan Drug designation to KB407, an investigational gene therapy for patients with cystic fibrosis (CF). More recently, at the start of 2023, the…
In recent years, treatment options for cystic fibrosis, a rare disease, have increased significantly in number and in effectiveness. However, many of the most effective treatments, developed by Vertex Pharmaceuticals,…
Newborn screening is a public health program in which infants are screened for various metabolic, genetic, and developmental disorders shortly after birth. Through newborn screening, doctors may identify potential disorders…
Proton pump inhibitors (PPIs) are a class of medicine that reduce the amount of stomach acid production. They are commonly known for their use in acid-related disorders, such as gastroesophageal…
CFRI's 35th Virtual National CF Education Conference: Focus on the Future July 29-31, 2022 CFRI’s National CF Education Conference brings together the national and international cystic fibrosis community for a…
Online Support Group for Teens with CF July 20th, 2022 This monthly group addresses the unique issues faced by teenagers between the ages of 13 and 18 growing up with…
Post-Transplant Support Group for Adults with CF July 27, 2022 This support group addresses the unique issues faced by those with CF who have received a double lung transplant and…
CF Caregivers Support Groups July 19, 2022 CFRI provides a monthly CF Caregivers Support Group, which is open to participants nationwide. The groups are facilitated by a social worker well versed…
Navigating Grief to Growth: Bereavement Support Group July 19th, 2022 A discussion and support group for those who have lost a loved one to CF, whether recently or in the…
Online Support Group for Adults with CF July 18th, 2022 CFRI provides a monthly Online Support Group for Adults with CF, which is open to participants nationwide. The group is facilitated by a…
Virtual Support Group for the Spanish-Speaking CF Community July 13th, 2022 This monthly group meets on the second Wednesday of every month and addresses the unique issues faced by Hispanic/Latinx…
Navigating Grief to Growth: Bereavement Support Group July 5th, 2022 A discussion and support group for those who have lost a loved one to CF, whether recently or in the…
Have you ever heard of azithromycin? It’s an antibiotic. Chances are, if you’ve ever had a bacterial infection, you’ve even taken it. But according to Medical XPress, researchers are currently…
Earlier this year, the MHRA in the United Kingdom expanded the license for Kaftrio, a treatment option for patients with cystic fibrosis (CF). When this expansion occurred, allowing for the…
Many of us are familiar with the famous story written by Roald Dahl titled Charlie and the Chocolate Factory; we know the tale of the golden ticket and Charlie's journey to…
It's safe to say that Gabe Barajas has never been afraid of a challenge. In fact, he embraces challenges - and the excitement and adventure they bring to his life.…
Cervical dysplasia, a precancerous condition, is defined as "the abnormal growth of cells on the surface of the cervix." While it does not typically cause symptoms, it does lead to…
Have you ever heard of a monocyte? Monocytes are a type of leukocyte, or white blood cell, which plays a role in immune response. These cells develop within our bone…
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…
On December 9, 2021, the National Organization for Rare Disorders (NORD) hosted a webinar program titled "Health Equity and Rare Disorders." This webinar was the first of a three-part series…
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…
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…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
