Second Only Case of “Growth Stunting” Rare Disease in Baby Girl

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Bricanna Brendel weighed just 4 pounds and 11 ounces when she was born on Sept. 10, 2013, to Travis Brendel and Lorie Hjelmstad after 34 weeks gestation. She has not grown much since birth. Bricanna was born with an extremely rare disease — only the second known case in the world — that is stunting her growth. Now 11 months old, she is only 24 inches long and weighs just 9 pounds. The disease is … [more here...]

With Nine Surgeries, A Delhi Woman Fights Von Hippel-Lindau

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Life cannot be more cruel to Payel Bhattacharya, a patient of Von Hippel-Lindau (VHL), a rare autosomal genetic condition that predisposes individuals … [more here...]

The Neuropathy Association to Host a “Rare Neuropathies: Getting Diagnosed, Getting Help” Facebook Chat

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In the U.S., the Rare Disease Act of 2002 defines a rare (or orphan) disease as any disease or condition affecting less than 200,000 people (or about … [more here...]

Trends in Rare Diseases. Why the “Ice Bucket Challenge” is a Cold Dash of Temporary Relief


Out of 14 different titles, Elsevier’s Cell Press has no Trends in Rare Diseases. To be fair, there are some rare diseases mentioned in some of the … [more here...]

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 … [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...]