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…
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…
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…
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