Widely Hailed Cystic Fibrosis Drug Orkambi Doesn’t Benefit All Patients
blickpixel / Pixabay

Widely Hailed Cystic Fibrosis Drug Orkambi Doesn’t Benefit All Patients

According to a story from Medical Xpress, a recently published French study evaluating the impact of the combination drug lumacaftor-ivacaftor (marketed as Orkambi) concluded that the drug had the potential…

Continue Reading Widely Hailed Cystic Fibrosis Drug Orkambi Doesn’t Benefit All Patients

An Experimental Cystic Fibrosis Treatment Could Make This Patient’s Greatest Wish Come True

According to a story from abc.net.au, Darren Bullock is getting pretty long in the tooth---at least for a cystic fibrosis patient. He is 44 years old, and his lungs currently…

Continue Reading An Experimental Cystic Fibrosis Treatment Could Make This Patient’s Greatest Wish Come True
A Tale of Two Sisters With Cystic Fibrosis Highlights the Barriers to Treatment Access That Still Remain
Free-Photos / Pixabay

A Tale of Two Sisters With Cystic Fibrosis Highlights the Barriers to Treatment Access That Still Remain

According to a story from the Sunday Post, sisters Shona and Kirsty Young, aged 24 and 29 years respectively, were both born with the devastating rare disease cystic fibrosis. However,…

Continue Reading A Tale of Two Sisters With Cystic Fibrosis Highlights the Barriers to Treatment Access That Still Remain
FDA Clears New Drug Application for an Experimental Triple Action Cystic Fibrosis Drug
Pexels / Pixabay

FDA Clears New Drug Application for an Experimental Triple Action Cystic Fibrosis Drug

According to a story from Market Watch, the biotechnology company Vertex Pharmaceuticals, Inc. has announced recently that the US Food and Drug Administration (FDA) has accepted the company's New Drug…

Continue Reading FDA Clears New Drug Application for an Experimental Triple Action Cystic Fibrosis Drug

Come On Pharma: It’s Not That Much of a Sacrifice to Study One Rare Disease Pro-Bono

At the heart of rare disease treatments is research. Currently, 95% of all rare diseases still have no approved therapies. Although there has been an increased focus on rare diseases…

Continue Reading Come On Pharma: It’s Not That Much of a Sacrifice to Study One Rare Disease Pro-Bono

Potential Treatment for Cystic Fibrosis Displays Encouraging Interim Data

According to a story from Drug Development Technology, the drug developer Translate Bio has recently reported interim results from its phase 1/2 clinical trial. This trial is testing the company's…

Continue Reading Potential Treatment for Cystic Fibrosis Displays Encouraging Interim Data

DNA Sequencing Took Years, Now it Takes Hours; As a Result, Researchers Have Discovered The Cause of Four Rare Diseases

Researchers at the University of Tokyo have discovered that one segment of DNA is responsible for four rare diseases. As reported in Science Daily, the university team also believes that…

Continue Reading DNA Sequencing Took Years, Now it Takes Hours; As a Result, Researchers Have Discovered The Cause of Four Rare Diseases
European Medicines Agency Grants Experimental Cystic Fibrosis Drug Orphan Drug Designation
source: pixabay.com

European Medicines Agency Grants Experimental Cystic Fibrosis Drug Orphan Drug Designation

According to a press release from Aridis Pharmaceuticals, the Company has received Orphan Drug designation from the European Medicines Agency for its experimental cystic fibrosis-related lung infection treatment AR-501. The…

Continue Reading European Medicines Agency Grants Experimental Cystic Fibrosis Drug Orphan Drug Designation

‘No Cure, No Pay’ Model in the Netherlands Means Patients Don’t Pay Unless Their Therapy Works

No Cure, No Pay Emile Voest, an oncologist in the Netherlands, conceived the idea of a no cure, no pay model. After witnessing the exorbitant cost of drugs, some of…

Continue Reading ‘No Cure, No Pay’ Model in the Netherlands Means Patients Don’t Pay Unless Their Therapy Works
Potential Treatment for Cystic Fibrosis Lung Infections Earns Orphan Drug Designation
geralt / Pixabay

Potential Treatment for Cystic Fibrosis Lung Infections Earns Orphan Drug Designation

According to a story from prnewswire.com, the biopharmaceutical company Aridis Pharmaceuticals, Inc. has recently announced that the US Food and Drug Administration (FDA) has awarded Orphan Drug designation to the…

Continue Reading Potential Treatment for Cystic Fibrosis Lung Infections Earns Orphan Drug Designation
With Time Running Out, an Artist With Cystic Fibrosis Reflects on Her Art and Experiences as a Patient
Monoar / Pixabay

With Time Running Out, an Artist With Cystic Fibrosis Reflects on Her Art and Experiences as a Patient

According to a story from BBC, 35 year old Kimberly Chard knows that she probably doesn't have much longer to live. Kimberly was born with cystic fibrosis, a rare disorder…

Continue Reading With Time Running Out, an Artist With Cystic Fibrosis Reflects on Her Art and Experiences as a Patient

Program Established by the NIH to Help Rare Disease Advocacy Groups Start Patient Registries

The Beginnings Anne Pariser worked for 16 years at the FDA. For part of that time, she was a team leader for the Center for Drug Evaluation and Research where…

Continue Reading Program Established by the NIH to Help Rare Disease Advocacy Groups Start Patient Registries
Close Menu