Rare Disease Day 2012 – Spread This Call To Action To Make A Difference!

World Rare Disease Day 2012 is coming up on February 29th.  Consider these staggering statistics. Each year, 139 BILLION U.S. dollars are invested in medical research — much of it from tax payers Each year, 800,000 papers are published in medical journals by scientists Each year on average, ONLY 21 new drugs are approved by […]

Why Vote4Hope? How Kickstarting Cures For Rare Disease Through The Pepsi Refresh Project Can Help Kids

Last week, I thought about trying to raise $250k in the Pepsi Refresh Project for The Addi and Cassi Fund and for Niemann Pick Type C disease research but being part of something larger that can make a difference for millions of ‘rare kids’ is important to me. That’s why I decided during the entire […]

A True Healthcare Reform Idea – Priority Review Vouchers For Ultra Rare Diseases

This week I have been reading all about priority review vouchers. Henry Grabowski and other faculty at Duke University came up with the idea for providing an incentive for drugmakers to target certain tropical and infectious diseases (such as TB, malaria, cholera, dengue, leprosy) affecting poor nations.  Senators Sam Brownback (R-KS) and Sherrod Brown (D-OH) […]

Denim Blue Jeans Ribbon To Symbolize Rare Disease Awareness; New Campaign of Jeans and Genes A Perfect Fit!

The Rare Disease community now has a powerful and universal new symbol to rally around — a blue jeans denim ribbon created by the Global Genes Project and the Children’s Rare Disease Network.  The worldwide initiative lauched today and is focused on raising awareness for millions of children living with rare and debilitating diseases like Addi and […]

Cystic Fibrosis and Niemann Pick Type C Linked Through Cholesterol Abnormalities and cAMP Cell Signaling Pathway

A few months ago, I wrote a blog about the connection points between Duchenne Muscular Dystrophy (DMD) and Niemann Pick Type C (NPC) and how more research dollars are needed for collaborative research projects between the two deadly diseases. Today, I am writing about the connections between Cystic Fibrosis (CF) and Niemann Pick Type C. […]