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…
The biotechnology company ContraFect has entered into an agreement with the Cystic Fibrosis Foundation. This funding agreement aims to investigate and develop direct lytic agents (DLAs) as a treatment for…
According to a story from england.nhs.uk, a recent agreement struck between the UK's NHS and Vertex Pharmaceuticals will herald a major improvement for the many of the country's cystic fibrosis…
Arrowhead Pharmaceuticals has began dosing patients in its AROENaC1001 trial, which evaluates ARO-ENaC as a treatment for cystic fibrosis. 54 patients will be treated with this therapy in an effort…
Three people with an eye on the future talk about their innermost feelings and the fact that their lives may be interrupted at any time by their chronic illness.…
According to a story from Youth Today, the Americans with Disabilities Act (ADA) was first passed 30 years ago, in 1990. This historic law helps protect people that live with…
At one point in his journey with cystic fibrosis, Marc Cotterill experienced a rapid loss in lung function. In fact, it dropped to just 29%. As his health worsened,…
According to a story from Cystic Fibrosis News Today, Eloxx Pharmaceuticals had just announced recently that it is officially re-opening enrollment for its phase 2 clinical study. This trial will…
Aridis Pharmaceuticals has released the data from the first phase of its clinical trial of AR-501. This treatment is inhaled to clear lung infections that are commonly experienced by those…
For the last few months, we've been inundated with stories of COVID-19, and with good reason, considering the global pandemic reached over 5 million cases worldwide as of this…
As reported in Medical Xpress, the U.S. Centers for Disease Control and Prevention have listed antibiotic resistance as one of the most pressing health challenges of our time. Infection treatment…
It's official: May is Cystic Fibrosis Awareness Month! So myself, and the Patient Worthy team, are excited to bring you some stories to help build your awareness and increase patient…
May is recognized annually as Cystic Fibrosis (CF) Awareness Month. Each year, this rare disease community comes together in order to help spread awareness about cystic fibrosis among the general…
Trevor O'Sullivan Trevor O'Sullivan, who lives in Ireland, has cystic fibrosis (CF). He has undergone two liver transplants. He was the 42nd person in the country to have this surgery.…
By Jodee Redmond from In The Cloud Copy At one time, hearing a cystic fibrosis (CF) diagnosis was tantamount to a death sentence for a young person. The median survival…
When the 31-year-old woman showed up to Massachusetts General Hospital with a nasty respiratory infection, she was somewhat used to it. In fact, she had been plagued with respiratory problems…
U.S. company, Vertex Pharmaceuticals, is the creator of three treatments for cystic fibrosis: Orkambi, Symdeco, und Kalydeco. These drugs were unavailable in Switzerland, as Vertex and Swiss health officials were…
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