Vertex’s 2-Drug Cystic Fibrosis Pill Shows Promise


Vertex Pharmaceuticals said on Tuesday that a combination of two of its drugs had successfully treated cystic fibrosis in closely watched clinical trials, potentially clearing the way for approval of a new option for nearly half the patients with the genetic disease. The drug combination, when compared with a placebo, improved the lung function of patients and also reduced pulmonary flare-ups, … [more here...]

I Gave My Stem Cells To Save a Life From Cancer And Now I Have Contracted a Deadly Disease With No Cure


My name is Hannah Heinzig, and I am 24 years old recently diagnosed with Pemphigus Vulgaris. This is how everyone starts out there story, so I will … [more here...]

Patient Advocacy Summit 2014: The Must-Have Collaborations for Successful Drug Development


Ready to hone your skills of collaboration and develop a better understanding of the drug development process? At this year’s Patient Advocacy Summit … [more here...]

Celiac to Refractory


On January 29th, 2009, I was hit head on by a drunk driver who was driving on the wrong side of the road. Within three months I became gravely ill. … [more here...]

September is Achalasia Awareness Month: How Will You Spread Awareness?


September is a particularly exciting month for Nancy Lazarus, founder of the Achalasia Awareness Organization. Lazarus was diagnosed in 1996 after … [more here...]

One Rare Disease Leads to Discovery of Another in Alexandra


Alexandra Mae Edges was born with Heterotaxy Syndrome and complex congenital heart defects. Heterotaxy Syndrome only occurs four our of every … [more here...]

Bangalore PhD Student on Coming Out with Thalassemia


My name is Namitha from Bangalore. I want to share my story to empower and enable others to come out as well. I am now a PhD student working on … [more here...]