Balancing Act Premieres New Segment on Paroxysmal Nocturnal Hemoglobinuria Tomorrow Morning

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(PNH) Paroxysmal Nocturnal Hemoglobinuria is a deadly life-threatening disease with devastating consequences.  35% of patients die within 5 years of diagnosis despite best supportive care.  You might not have heard of PNH – it’s a rare blood disorder that affects a few patients per million.  But its rarity doesn’t make it any less significant.  Join one Dr. Eloy Roman, one of the country’s only … [more here...]

3rd Annual Orphan Drugs and Rare Diseases Conference Taking Place Oct 20-21st in London

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SMi are proud to present the 3rd Annual Orphan Drugs and Rare Diseases Conference. The Orphan Drugs market is set to rise as a result of … [more here...]

A Family Diagnosis of Adult Polyglucosan Body Disease (APBD)


I was in my early 50's when I started experiencing leg cramps, stiffness and heaviness in my legs, muscle twitching and soreness, foot dragging, and … [more here...]

Las Vegas Art2 Downtown Arts Festival Held in Honor of GBS-CIDP International Foundation


The Art² Downtown Arts Festival with be a one-day art festival celebrating the best independent artist, craftsmen, and designers from around the Las … [more here...]

“How Can We Incorporate the Patient’s Voice?” Mass. Biotech Hosts Conference on Nov 10th

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The Massachusetts Biotechnology Council is hosting a conference this November 11th, 2015. The organization is an association of more than 650 … [more here...]

The Daily Pilot Reports on Global Genes 2014 Gala


Here are some sobering facts. Despite all of the advances in science and medicine, more than 7,000 types of rare diseases affect the human … [more here...]

Rare Disease Report: Five Myths That Slow Down Clinical Trial Enrollment

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Melissa Hogan, founder of Saving Case – an organization designed to help raise awareness of Hunter syndrome and engage people in the rare disease … [more here...]