Vertex’s 2-Drug Cystic Fibrosis Pill Shows Promise

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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

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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

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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

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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?

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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

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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

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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...]