FDA Does Not Approve Actelion’s Zavesca in US – Distressing News For Niemann Pick Type C Community
March 9, 2010 by Chris Hempel
Filed under Featured Stories
Actelion, the makers of Zavesca, today put out a brief statement saying that the U.S. Food and Drug Administration did not approve Zavesca and wants more information from the company on treating Niemann Pick Type C, a rare and dementing neurological disease that afflicts our six year old identical twins, Addi and Cassi. Niemann Pick Type C disease is caused […]
The Road to Building a New Orphan Drug – Am I Mad?
February 4, 2010 by Chris Hempel
Filed under Cyclodextrin
On February 25-26, 2010, I will be attending the inaugural FDA Build-an-Orphan-Drug-Workshop hosted by the Center for Rare Disease Therapies at Keck Graduate Institute (KGI) in Claremont, Calif. FDA agency experts will be on hand to provide guidance on applying for orphan drug designation. I will be attending the workshop with Dr. Ron Browne, hired […]
American Heart Association Features Addi and Cassi’s Fatal Genetic Cholesterol Disorder
February 1, 2010 by Chris Hempel
Filed under Featured Stories
Thank you to the American Heart Association for featuring Addi and Cassi Hempel’s story and increasing awareness of Niemann Pick Type C, an ultra rare and fatal cellular cholesterol disease that is trying to steal our twins away from us. We hope that by reading our story, more people will understand the significance of the Niemann Pick Type C […]
Nothing Bundt Cakes for Nothing Bundt Beautiful Birthday Girls
January 26, 2010 by Chris Hempel
Filed under Featured Stories
Addi and Cassi turned six years old on Saturday. Over two years ago when the twins were diagnosed with Niemann Pick Type C disease, I was not sure they would still be with us today. Our little angels are amazing fighters and doing very well on their cyclodextrin infusions. Here is the ice cream cone […]
Family of Twins with Rare and Fatal Cholesterol Disease To Seek FDA Approval To Deliver Cyclodextrin Into the Brain
January 15, 2010 by Chris Hempel
Filed under Cyclodextrin, Cyclodextrin News
The tagline of the new movie, Extraordinary Measures, about John Crowley’s fight to save his children from the rare and fatal Pompe disease is….Don’t Hope For a Miracle – Make One! We are now working on making our own miracle – a new compassionate use investigational new drug (IND) protocol and application that we plan […]
