FDA Filing Made Requesting Use of Medtronic SynchroMed Pump To Deliver Cyclodextrin To Brain
April 13, 2011 by Chris Hempel
Filed under Featured Stories
To say I am pumped up today is an understatement! We’ve reached another historic milestone in our effort to treat Addi and Cassi with cyclodextrin! Dr. Caroline Hastings at Children’s Hospital Research Center Oakland filed a 400+ page document with the FDA yesterday on behalf of the twins seeking a permanent solution to deliver hydroxyl-propyl-beta-cyclodextrin [...]
National Center for Advancing Translational Sciences (NCATS) Announced By National Institutes of Health (NIH) Director Francis Collins
March 9, 2011 by Chris Hempel
Filed under Featured Stories
The Global Genes Project, a rare disease patient advocacy organization, was invited to present a giant handmade denim genes ribbon to Dr. Francis Collins on February 28 — Rare Disease Day 2011. This year the NIH Office of Rare Disease Research (ORDR) encouraged everyone to wear jeans to show support for the campaign of using [...]
National Institutes Of Health to Host Rare Disease Day Event; Dr. Francis Collins To Speak; Wear Jeans!
January 9, 2011 by Chris Hempel
Filed under Featured Stories
The National Institutes of Health (NIH) will celebrate the 4rd Rare Disease Day on February 28, 2011, with a day-long celebration and recognition of the various rare diseases research activities. The event will be held in the Lipsett Amphitheater from 8:30am to 5:15pm. Dr. Francis Collins is expected to speak. A tentative agenda has been [...]
FDA Approves Request For New Cyclodextrin Treatment For Niemann Pick Type C
September 25, 2010 by Chris Hempel
Filed under Cyclodextrin, Featured Content
FDA Approval Received! Children’s Hospital Oakland Receives FDA Clearance to Begin World’s First Cyclodextrin Administration Into the Brains of Twins with Rare and Deadly Cholesterol Disease Sugar Molecule Used In Common Food and Household Products Like Febreze® Fabric Refresher Called Hydroxypropyl Beta Cyclodextrin (HPßCD) Will be Delivered into Twins’ Central Nervous System in an Attempt [...]
Why Vote4Hope? How Kickstarting Cures For Rare Disease Through The Pepsi Refresh Project Can Help Kids
August 30, 2010 by Chris Hempel
Filed under Featured Stories
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 [...]
