Historically, drug development within the rare disease space has been stifled; companies tend to focus on developing therapies for bigger patient populations that will be more impactful and more…
Nasal polyps are non-cancerous growths that develop into the nasal passages. Generally, they don't cause any pain. However, when they reach a certain size, they can inhibit the sense of…
65 roses. This phrase is often used in reference to cystic fibrosis; in fact, the origin of 65 roses is a young boy who couldn’t properly pronounce the name…
Findings from the Max Delbrück Center and the Charité Universitätsmedizin Berlin have been published in the European Respiratory Journal stating that a triple combination therapy can improve CF symptoms…
According to a story from European Pharmaceutical Review, the European Commission has recently approved a label extension of ORKAMBI (lumacaftor/ivacaftor), a drug used to treat cystic fibrosis. Under the extension,…
Advances in research and medicine have improved the life expectancy for people with cystic fibrosis. But this doesn't mean that we should stop searching for a cure. Most likely,…
“Three years ago, my lung function was 11% and I was hours from death, then I received the gift of life – which was Trikafta.” These are the words of…
There is no cure for cystic fibrosis. That is the reason the Cystic Fibrosis Research Institute (CFRI) is very vocal and makes it clear that cooperation is needed from the…
Treatment advances are occurring daily in the field of rare disease. As researchers hone their strategies and tools, new technologies are emerging with the potential to significantly impact patients…
Rylee Riekena feels most at home when she’s near water. There’s something about the ocean that just makes her feel safe and at peace. So when she took up paddleboarding,…
Each year, students at Carteret Community College in Morehead City, North Carolina participate in the “Great Strides” Walk for Cystic Fibrosis. “Great Strides” is the Cystic Fibrosis Foundation’s largest fundraising…
Boshamps Seafood & Oyster House has been serving Destin, FL with authentic Gulf seafood, well-mixed libations, and live music for years. But that isn’t the only way Boshamps has made…
Children with cystic fibrosis (CF) born between 1995 and 1999 had an estimated life expectancy of 32 years. Advances in both research and treatment have contributed to increased life…
The advocacy group Just Treatment recently published a press release titled 'BREAKING: Cystic Fibrosis Patients Launch Global Challenge to Vertex Monopoly on CF Drugs.' Vertex Pharmaceuticals has taken the lead…
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…
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”…
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