Friday, July 30, 2010

Featured Stories

Alzheimer’s Patients Presenilin 1 Gene Defect Causes A Lysosomal Storage Disorder Alzheimer’s Patients Presenilin 1 Gene Defect Causes A Lysosomal Storage Disorder

A research paper by Dr. Ralph Nixon, director of the Center of Excellence on Brain... 


Orphan Drug Act  – A Collosal Failure Considering Rare Disease Drug Statistics Orphan Drug Act – A Collosal Failure Considering Rare Disease Drug Statistics

For the past week, I have been obsessing over rare disease statistics ever since... 


New FDA ‘Rare Disease’ Division Being Proposed to U.S. House Committee on Appropriations New FDA ‘Rare Disease’ Division Being Proposed to U.S. House Committee on Appropriations

The Kakkis Every Life Foundation is leading the effort to request $10 million in... 


FDA Grants Orphan Drug Status For Cyclodextrin Compound To Treat Fatal Genetic Cholesterol Disease FDA Grants Orphan Drug Status For Cyclodextrin Compound To Treat Fatal Genetic Cholesterol Disease

Drum roll, please! Dr. Caroline Hastings at Children’s Hospital Oakland and... 


SeizureTracker – Great Tool For Tracking and Recording Seizures SeizureTracker – Great Tool For Tracking and Recording Seizures

This week I started using an online program called SeizureTracker. Over the past... 


Dementia in Children and Teens – When Kids Brains Regress Like The Elderly Dementia in Children and Teens – When Kids Brains Regress Like The Elderly

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... 


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


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Cyclodextrin

IND Application Filed With FDA To Bring Cyclodextrin Into The Brain To Treat Fatal Childhood Cholesterol Disease IND Application Filed With FDA To Bring Cyclodextrin Into The Brain To Treat Fatal Childhood Cholesterol Disease

After months and months of work and input by doctors and researchers, Dr. Caroline... 


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... 


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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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