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)

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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

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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

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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...]