Friday, March 12, 2010

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FDAs Orphan Drug Workshop Featured in Wall Street Journal – Push To Cure Rare Diseases

Below is a story that ran in today’s Wall Street Journal (Page A 3) on the... 

FDA Does Not Approve Actelion’s Zavesca in US – Distressing News For Niemann Pick Type C Community FDA Does Not Approve Actelion’s Zavesca in US – Distressing News For Niemann Pick Type C Community

Addi and Cassi's monthly dose of the drug Zavesca made by Actelion Actelion,... 

Obama Cracks Jokes About Cholesterol Level And Personal Health – Is He Out of Touch? Obama Cracks Jokes About Cholesterol Level And Personal Health – Is He Out of Touch?

I almost had a heart attack today (really, no pun intended) when I read that President... 

FDA and EMA Forge Rare Disease Collaboration; Announcement Coincides with World Rare Disease Day 2010 FDA and EMA Forge Rare Disease Collaboration; Announcement Coincides with World Rare Disease Day 2010

In recognition of World Rare Disease Day 2010, the U.S. Food and Drug Administration... 

FDA Orphan Drug Workshop Makes Filing Applications Easy – Even For Rare Disease Patient Advocates! FDA Orphan Drug Workshop Makes Filing Applications Easy – Even For Rare Disease Patient Advocates!

Our FDA Orphan Drug Filing For Cyclodextrin To Treat Niemann Pick Type C Disease At... 

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

Numbers Don’t Add Up – Only 347 New Drugs Approved By FDA For 30 Million Americans With Rare Disease in Past 25 Years? Numbers Don’t Add Up – Only 347 New Drugs Approved By FDA For 30 Million Americans With Rare Disease in Past 25 Years?

When it comes to finding Rare Disease statistics, the same old statistics and facts... 

American Heart Association Features Addi and Cassi’s Fatal Genetic Cholesterol Disorder American Heart Association Features Addi and Cassi’s Fatal Genetic Cholesterol Disorder

Thank you to the American Heart Association for featuring Addi and Cassi Hempel’s story... 

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Cyclodextrin

The Road to Building a New Orphan Drug – Am I Mad? The Road to Building a New Orphan Drug – Am I Mad?

On February 25-26, 2010, I will be attending the inaugural FDA Build-an-Orphan-Drug-Workshop... 

Family of Twins with Rare and Fatal Cholesterol Disease To Seek FDA Approval To Deliver Cyclodextrin Into the Brain Family of Twins with Rare and Fatal Cholesterol Disease To Seek FDA Approval To Deliver Cyclodextrin Into the Brain

The tagline of the new movie, Extraordinary Measures, about John Crowley’s fight... 

Instructions On How Cyclodextrin Solution Is Made For Addi and Cassi’s Intravenous Cyclodextrin Therapy Instructions On How Cyclodextrin Solution Is Made For Addi and Cassi’s Intravenous Cyclodextrin Therapy

A number of people from around the world who have children diagnosed with the fatal... 

Parents To Seek FDA Orphan Drug Designation For Cyclodextrin To Treat Rare and Fatal Cholesterol Disease Parents To Seek FDA Orphan Drug Designation For Cyclodextrin To Treat Rare and Fatal Cholesterol Disease

On Tuesday morning at 11am, Addi and Cassi are going to undergo volumetric CT scans... 

FDA Investigational New Drug Application Documents for Cyclodextrin Treatment for Niemann Pick Type C Disease FDA Investigational New Drug Application Documents for Cyclodextrin Treatment for Niemann Pick Type C Disease

Here you will find detailed documents outlining Addi and Cassi’s cyclodextrin... 

New non-drug fix for HIV? New non-drug fix for HIV?

The Scientist Magazine By Alison McCook June 2009 Researchers are slowly establishing... 

Wacker Chemie Expands US Based Cyclodextrin Facility – Sugar Compound Poses No Known Health Risks Wacker Chemie Expands US Based Cyclodextrin Facility – Sugar Compound Poses No Known Health Risks

Wacker Chemie, the Munich-based chemical company, announced that is has expanded... 

Anthrax Bacteria Killed By Simple Sugar Compound Called Cyclodextrin. Is CDC Looking Into This? Anthrax Bacteria Killed By Simple Sugar Compound Called Cyclodextrin. Is CDC Looking Into This?

We all remember the Anthrax-laced letters that killed five people and severely rattled... 

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Videos

University of Notre Dame Airs Video During Football Game Highlighting Rare Cholesterol Disease University of Notre Dame Airs Video During Football Game Highlighting Rare Cholesterol Disease

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]

CNN Covers How California Health Care Budget Cuts Threaten Life of Little Girl Suffering From Deadly Cholesterol Disease CNN Covers How California Health Care Budget Cuts Threaten Life of Little Girl Suffering From Deadly Cholesterol Disease

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]

What Dying Kids Can Teach Our Government About Health Care Reform What Dying Kids Can Teach Our Government About Health Care Reform

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]

Extra TV Renegade Doctors Show Covers FDA Approval of Addi and Cassi’s Cyclodextrin Infusions Extra TV Renegade Doctors Show Covers FDA Approval of Addi and Cassi’s Cyclodextrin Infusions

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]

Mom Finds Experimental Drug for Twins’ Fatal Cholesterol Condition; FDA Approves Treatment Under Compassionate Use Program Mom Finds Experimental Drug for Twins’ Fatal Cholesterol Condition; FDA Approves Treatment Under Compassionate Use Program

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]

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