Monday, May 25, 2020

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Drug for Rare Disorder Linked to Reducing Cholesterol Plaque Drug for Rare Disorder Linked to Reducing Cholesterol Plaque

Compound being tested in a genetic condition showed promise in treating heart disease... 

In Science, Inspiration Can Come From Unlikely Places In Science, Inspiration Can Come From Unlikely Places

A mom reached out to a scientist with a novel idea for an experiment. Years later,... 

CNN – Dr. Sanjay Gupta Chronicles Our Fight CNN – Dr. Sanjay Gupta Chronicles Our Fight

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NPC Disease and it’s Connection to Ebola NPC Disease and it’s Connection to Ebola

Amy Marcus at the Wall Street Journal has written a short story about the unexpected... 

Addi & Cassi Featured in an Incredible Wall Street Journal Story Addi & Cassi Featured in an Incredible Wall Street Journal Story

Pulitzer prize winning journalist Amy Marcus spent 6 years following Addi & Cassi,... 

Children’s Cancer Clinic Opens in Reno Led By Pediatric Hematologist Oncologist Dr. Caroline Hastings (Addi and Cassi’s doctor!) Children’s Cancer Clinic Opens in Reno Led By Pediatric Hematologist Oncologist Dr. Caroline Hastings (Addi and Cassi’s doctor!)

Dr. Caroline Hastings with Addi and Cassi's experimental medicine called cyclodextrin Many... 

Rare Disease Day Logo and Ribbons – Patient Advocates Join The RARE Team! Rare Disease Day Logo and Ribbons – Patient Advocates Join The RARE Team!

Here are the Rare Disease Day logos for 2012 from the Global Genes Project, the leading... 

Is Your Disease on the RARE List™ – If So, More Bad News! Is Your Disease on the RARE List™ – If So, More Bad News!

I wonder if people truly understand what it means if their rare disease or disorder... 

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Cyclodextrin

Walgreen’s Support Allows Our Twins with Niemann Pick Type C To Receive Cyclodextrin Treatments At Home Walgreen’s Support Allows Our Twins with Niemann Pick Type C To Receive Cyclodextrin Treatments At Home

Addi (l) and Cassi (r) will start their IV Cyclodextrin treatments at home next week... 

FDA Approves Request For New Cyclodextrin Treatment For Niemann Pick Type C FDA Approves Request For New Cyclodextrin Treatment For Niemann Pick Type C

Cyclodextrin Infusion bottles - We'll now mix cyclodextrin in saline and put... 

Cyclodextrins – Recipes To Make Edible Nanostructures and CD MOFs To LifeSaving Drug Treatments Cyclodextrins – Recipes To Make Edible Nanostructures and CD MOFs To LifeSaving Drug Treatments

Kenneth Chang of the New York Times reports on researchers creating edible nanostructures... 

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... 

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

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Videos

CNN – Dr. Sanjay Gupta Chronicles Our Fight CNN – Dr. Sanjay Gupta Chronicles Our Fight

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A Mother’s Quest – Dateline NBC Story 2009

DATELINE Covers FDA Approval of Twins Cyclodextrin Infusions  Read More →

Vote 4 Hope at Pepsi Refresh To Find Cures for Rare Skin Conditions like Epidermolysis Bullosa, Harlequin Ichthyosis, Xeroderma-Pigmentosum Vote 4 Hope at Pepsi Refresh To Find Cures for Rare Skin Conditions like Epidermolysis Bullosa, Harlequin Ichthyosis, Xeroderma-Pigmentosum

During the Vote4Hope Pepsi Refresh Rare Disease Campaign, I am making video postings throughout September to raise awareness for all rare diseases, not simply the horrific disease called Niemann Pick Type C that my six year old identical twins suffer from. Today’s videos are some of the most heartwrenching videos I have ever watched. The videos... [Read more of this review]

Rare Disease Foundations Need To Vote 4 Hope In Pepsi Refresh Rare Disease Foundations Need To Vote 4 Hope In Pepsi Refresh

During the Vote4Hope Pepsi Refresh Rare Disease Campaign, I am making video postings on Addi and Cassi’s website to raise awareness for all rare diseases, not just the horrific disease called Niemann Pick Type C that I am fighting against on behalf of my six year old identical twins. Please take a minute to watch this compelling rare disease video... [Read more of this review]

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]

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