Drug for Rare Disorder Linked to Reducing Cholesterol Plaque
Compound being tested in a genetic condition showed promise in treating heart disease in mice By: Amy Dockser Marcus (Wall Street Journal) Updated April 6, 2016 4:19 p.m. ET Cyclodextrin, a compound now in testing to treat a very rare genetic disease, may have a potential use in treating a much more common ...


In Science, Inspiration Can Come From Unlikely Places
A mom reached out to a scientist with a novel idea for an experiment. Years later, her hunch has blossomed into a published scientific study. By: Amy Dockser Marcus (Wall Street Journal) Updated April 6, 2016 2:08 p.m. ET In science, ideas can come from anywhere. Still, Eicke Latz was surprised to get ...


Addi & Cassi Featured in an Incredible Wall Street Journal Story
Pulitzer prize winning journalist Amy Marcus spent 6 years following Addi & Cassi, their doctors, and the science community on our journey to find treatments. In an unprecedented 10 chapter online e-book, the Wall Street Journal has published this remarkable story. The story (without the amazing videos, audioclips) is featured on ...


Featured Stories

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

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


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

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

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

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

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

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

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

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

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...
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Videos

A Mother’s Quest – Dateline NBC Story 2009
DATELINE Covers FDA Approval of Twins Cyclodextrin Infusions Read More →

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]

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]

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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