FDA Approves First Ever Cyclodextrin Infusion Treatment For Twin Girls Suffering From Fatal Cholesterol Disease
After months of research, document preparation, discussions with the FDA and an immense amount of stress, it's finally official. The FDA has approved our "compassionate use" IND request to give Addi and Cassi infusions of a non toxic sugar compound called 2-Hydroxypropyl-beta-Cyclodextrin or HPBCD. This Wednesday, March 18, 2009, at 7:30am and 8:30am, ...
Join Global Genes Project and Get Your Denim Ribbon To Support World Rare Disease Day 2010
World Rare Disease Day 2010 will be held on February 28. Please join the Global Genes Project to help raise awareness for Rare Diseases of all types by wearing denim on Feb. 28 or holding an event in your local area to raise money for your favorite Rare Disease charity ...
The Wall Street Journal Reports On FDA Approval of Addi and Cassi’s Cyclodextrin Treatment
By AMY DOCKSER MARCUS April 3, 2009 A Mom Brokers Treatment for Her Twins' Fatal Illness Bucking Scientific Convention, Ms. Hempel Gets Researchers From Different Fields to Share Data on Potential Therapy From the moment her twin daughters, Addison and Cassidy, were diagnosed with a fatal genetic disease ...
Ten Tips To Start Your Own Virtual BioTech To Find Treatments For Your Rare Disease
Ever since we went public with Addi and Cassi’s cyclodextrin treatments, I have received countless emails asking me a number of questions. How did I find the sugar compound "cyclodextrin?" How did I manage to get the Food and Drug Administration to approve infusions of this new compound into Addi ...
Life Versus Freezers
If you were a frozen embryo, would you rather die in a freezer or donate your life to science? It seems like a very easy choice to make, don't you think? I have never had to think much about the stem cell debate before Addi and Cassi were diagnosed with ...
CureCaps – A Global Symbol Of Hope
Volunteer To Make CureCaps - Hand in Hand We Will Find Cures For The Brain In November 2007, we organized a grassroots effort called CureCaps and asked volunteers from around the world to help us fight deadly neurological diseases like Niemann Pick Type C by making hats to raise money for ...
Featured Stories
A research paper by Dr. Ralph Nixon, director of the Center of Excellence on Brain...
For the past week, I have been obsessing over rare disease statistics ever since...
The Kakkis Every Life Foundation is leading the effort to request $10 million in...
Drum roll, please! Dr. Caroline Hastings at Children’s Hospital Oakland and...
This week I started using an online program called SeizureTracker. Over the past...
The Alzheimer’s Society and Alzheimer’s Association have lots of information...
Guidelines To Write and Submit an Orphan Drug Application For A Rare Disease
While working on our orphan drug application for Cyclodextrin for the treatment of...
Below is a story that ran in today’s Wall Street Journal (Page A 3) on the...
Read More Posts From This Category
Cyclodextrin
After months and months of work and input by doctors and researchers, Dr. Caroline...
On February 25-26, 2010, I will be attending the inaugural FDA Build-an-Orphan-Drug-Workshop...
The tagline of the new movie, Extraordinary Measures, about John Crowley’s fight...
A number of people from around the world who have children diagnosed with the fatal...
On Tuesday morning at 11am, Addi and Cassi are going to undergo volumetric CT scans...
Here you will find detailed documents outlining Addi and Cassi’s cyclodextrin...
The Scientist Magazine By Alison McCook June 2009 Researchers are slowly establishing...
Wacker Chemie, the Munich-based chemical company, announced that is has expanded...
Read More Posts From This Category
Videos
Thanks to the University of Notre Dame for highlighting the Parseghian Family’s fight against Niemann Pick Type C disease during the Notre Dame versus University of Nevada Reno football game. They have a true commitment to helping fight not only Niemann Pick Type C but rare disease of all types. A number of people in the Reno area who are... [Read more of this review]
Embedded video from CNN Video Here is a story on sweet little Jessica Leoni who suffers from the same disease as Addi and Cassi. Her health care is being threatened by California’s Health Care crisis. Here is the story from CNN. LOS ANGELES, California (CNN) — Anthony and Lisa Leoni have little time to worry about whether California’s... [Read more of this review]
Addi & Cassi Story II from Addi & Cassi Hempel on Vimeo. Here is a video we put together for Oprah last year about the issues we are facing with health care and drug development system in the United States and what happens when your children are dying from a rare and fatal cholesterol disease like Niemann-Pick Type C (NPC). This video was... [Read more of this review]
Renegade Doctors (Extra) Story about Addi & Cassi Hempel’s Infusions of Cyclodextrin from Addi & Cassi Hempel on Vimeo. Twins Addison and Cassidy Hempel are fighting a disease that steals their memory, but they’re only five years old. Now their mother Chris is on a crusade to stop Niemann-Pick Disease Type C, a disease similar... [Read more of this review]
Good Morning America reports on the Food and Drug Administration’s approval of Addi and Cassi’s compassionate use Investigational New Drug Application to give them intravenous infusions of cyclodextrin, a non toxic sugar compound found in everyday food products. Cyclodextrin may have the ability to remove trapped cellular cholesterol from... [Read more of this review]
Read More Posts From Videos





