Rare Disease Patient Advocacy Summit
Later this week, Jeanne Barnett will be attending the fifth annual RARE Patient Advocacy Summit run by Global Genes in Huntington Beach, California. The goal of this year’s RARE Patient Advocacy Summit is for patients, caregivers, and advocates to walk away equipped with actionable next steps whether you have been …
Barnyard Dust and Preventing Asthma in Children (one of many Cystic Fibrosis symptoms)
People with cystic fibrosis have infection and inflammation in their lungs, and some also have problems with asthma, making asthma one of the many cystic fibrosis symptoms that patients experience. From the CDC: the prevalence of asthma in the general population of the U.S. is 7-9%, and as many as …
SuperHero DNA
Study finds individuals immune to severe diseases. A new study has found that certain people’s genes have evolved in such a way that they are immune to certain severe inherited conditions. The study involved the analysis of previously collected DNA sets totaling 589,306 genomes from around the world with researchers hoping …
Surprising Benefits from CF Research
CF Research May Help Agriculture Cystic Fibrosis is often referred to as an orphan disease i.e. research benefits a relatively small portion of the general population. But here is a serendipitous situation in which CF research may help agriculture on a large scale. A graduate student at the University of …
UCSD’s 3D Printed Liver Cells Most Liver-like Yet
3 D Printed Liver Image from ccsmobile.co.uk Feb 9, 2016 Home to the original bioprinting firm, Organovo, San Diego is, apparently, a hotbed of bioprinting activity. Or, at the very least, the University of California, San Diego is make its own strides in the field of bioprinted liver tissue. Published …
CF Patient To Climb the UK’s Three Highest Mountains in 24 Hours
Written by Leonor Mateus Ferreira Source: Cystic Fibrosis News Today Not long after his birth in 2013, Paul Siebenthal‘s son Noah was diagnosed with cystic fibrosis (CF) and the new father became aware that his son would struggle with a chronic, life-threatening disease for his entire life. However, Paul Siebenthal did not accept that …
New cystic fibrosis drug has high potential, high cost
Written by Tracey Walker Source: Managed Healthcare Executive FDA’s approval of Vertex Pharmaceutical’s groundbreaking lumacaftor/ivacaftor (Orkambi) for cystic fibrosis (CF) is the first drug to treat the underlying cause of CF in people ages 12 and older with two copies of the F508del mutation. CF is an inherited chronic disease that affects …