Drug for Rare Disorder Linked to Reducing Cholesterol Plaque
April 6, 2016 by Daddy
Filed under Featured Content, Featured Stories
Compound being tested in a genetic condition showed promise in treating heart disease in mice By: Amy Dockser Marcus (Wall Street Journal) Updated April 6, 2016 4:19 p.m. ET Cyclodextrin, a compound now in testing to treat a very rare genetic disease, may have a potential use in treating a much more common condition too: […]
In Science, Inspiration Can Come From Unlikely Places
April 6, 2016 by Daddy
Filed under Featured Content, Featured Stories
A mom reached out to a scientist with a novel idea for an experiment. Years later, her hunch has blossomed into a published scientific study. By: Amy Dockser Marcus (Wall Street Journal) Updated April 6, 2016 2:08 p.m. ET In science, ideas can come from anywhere. Still, Eicke Latz was surprised to get an email […]
Walgreen’s Support Allows Our Twins with Niemann Pick Type C To Receive Cyclodextrin Treatments At Home
July 8, 2012 by Chris Hempel
Filed under Cyclodextrin
We are sorry for the lack of posts in recent months! Hugh and I have been extremely busy with rare disease initiatives for Solution Therapeutics, our virtual biotech, as well as helping the Global Genes Project with a number of community based rare disease projects. We would like to report that after three years of […]
From The Valley of Death to The Valley of Cures – NIH TRND Program Supports Multiple Rare Diseases
November 15, 2011 by Chris Hempel
Filed under Featured Stories
Thank you Dr. Francis Collins, Dr. Chris Austin, Dr. John McKew and others at the NIH who are all involved in driving the National Institutes of Health’s Therapeutics for Rare and Neglected Diseases (TRND) program forward. TRND has been a huge success since launching five pilot projects last year, including one program that focuses on […]
National Institues of Health Announces Cyclodextrin Clincial Trial For Fatal Cholesterol Disease Called Niemann Pick Type C
August 16, 2011 by Chris Hempel
Filed under Featured Stories
Over the past three years, many Niemann Pick Type C families as well as physicians treating NPC patients worldwide have contacted Dr. Caroline Hastings and I regarding Addi and Cassi’s treatments with hydroxypropyl-beta-cyclodextrin. Since sharing our intravenous (IV) and intrathecal (IT) cyclodextrin treatment protocols with NPC families and doctors in the U.S. and other countries, […]
