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…
Selma Santos has met many nurses over the course of her life. Having a daughter with cystic fibrosis means spending a lot of time in the hospital or at doctor's…
Dan Hopkins is a bowler who has recently raised over $21,000 for cystic fibrosis (CF) in a 65 hour long bowl-a-thon. While he may be exhausted and sore, he is…
On November 27, biotechnology company Vertex Pharmaceuticals Inc. ("Vertex") announced the approval of SYMKEVI with KALYDECO for the treatment of pediatric patients with cystic fibrosis (CF), says GuruFocus. The…
As reported in Warta Saya; for kids with cystic fibrosis (CF), a daily exercise needed to clear the lungs of the disease's characteristic build-up of mucus can feel long and…
Bulgarian cystic fibrosis patient joins international campaign standing up for the rights of patients to access essential medicines Rositsa Malinova, a 22-year-old resident of Pazardzhik, Bulgaria, is taking on the…
Transitions to adult care for patients who have a chronic respiratory disease, like cystic fibrosis, or a type of neuromuscular disease that influences the respiratory system is something that the medical…
Maintaining lung function is a major goal of cystic fibrosis (CF) treatment. In order to accomplish this goal, doctors will use airway clearance techniques, such as vest therapy. The problem…
Lung infections are a common symptom of cystic fibrosis, with Staphylococcus aureus being the most prevalent. While physicians can treat this infection using antibiotics, they do not yet understand its role…
by Danielle Bradshaw from In The Cloud Copy A recently performed study has demonstrated that it is possible to use tablets to screen patients that have chronic lung conditions -…
In late September and early October, the North American Cystic Fibrosis Conference (NACFC) went virtual, as many conferences and symposia this year have been forced to do. However, that does…
According to a story from BioSpace, the US Food and Drug Administration (FDA) recently announced that it has awarded grant funding that will go towards six clinical trials that will…
Later this week, from October 21-23, presenters and attendees will be present at the 34th Annual North American Cystic Fibrosis Conference (NACFC). Although this year is slightly different, considering the…
The United States FDA has just announced that they will be providing 6 new grants, which are worth 16 million dollars for rare disease research. These grants will last a…
Happy Friday! October is going by quickly, but if you need something else to help pass the time, why not give our podcast a listen? The two newest episodes of,…
Ionis Pharmaceuticals has recently released the data from their clinical trial of IONIS-ENAC-2.5Rx, a treatment for cystic fibrosis. According to Newswire, the study showed very positive results, with healthy volunteers…
As reported in Cystic Fibrosis News; the 2020 Santander Universities Entrepreneurship Awards are part of an initiative to encourage young entrepreneurship via support of student led startups. This year there…
The FDA has recently granted two designations, the rare pediatric disease and orphan drug designations, to Spirovant Sciences' gene therapy for cystic fibrosis (CF). SPIRO-2101 will now be able to…
Vertex Pharmaceuticals will present data on the long-term use of their cystic fibrosis (CF) treatments at the 43rd European Cystic Fibrosis Digital Conference, and then again at the 2020 North…
On September 25, biotechnology company Vertex Pharmaceuticals Incorporated ("Vertex") announced FDA-approval of KALYDECO (ivacaftor) for pediatric patients with cystic fibrosis (CF). Specifically, KALYDECO can treat infants between 4-6 months…
According to a story from BioSpace, the biopharmaceutical company Aridis Pharmaceuticals, Inc., has recently announced that it has struck an agreement with the US Food and Drug Administration (FDA) in…
As reported in the Global News; what if you had a draining rare disease, and a life-changing treatment was out there but wasn't available in your country? At Dalhousie University…
© 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.
