A Global Fight Against Cholesterol and Dementia

Kaitlyn Maher Sings “You Were Meant To Be” Kids Suffering from Niemann Pick Type C disease, the “Childhood Alzheimer’s.” Approximately 500 children around the world and of all ethnic backgrounds are afflicted with Niemann Pick Type C disease.

We hope this video and pictures of these precious children will shed some light as to why we need your donations to find a cure for Niemann Pick Type C.

• Peyton and Kayla Hadley, Medford, Oregon (Hadley Hope Fund)
• Hope For Aaditya – India
• Riko Mizusawa – Japan
• Brisan and Parker Stults, Kansas City, MO
• Gabrielle Laverde, Deltona, Florida, USA
• Dana and Andrew Marella (Dana’s Angels Research Trust – DART)
• Adam Recke, Allentown, PA, USA (Race For Adam)
• Dillon Papier, Urbana, Maryland
• Matt and Tim Lloyd, Australia
• Elise Barton, Minneapolis, Minnesota
• Jessica Leoni, Los Angeles, California
• Hope For Hollie, United Kingdom
• Mindy Fagan, Michigan, USA
• Tylor White Richardson, Duboque, Iowa, USA
• Lois Peacock, UK
• Kure For Kirkland, Toronto, Canada
• Gage Luskey, Edmonton, Alberta, Canada
• Taylor Smith, Barrow, United Kingdom
• Calum Burdon, United Kingdom
• Ty Quandt, Washington, USA
• Mindy Fagan, Michigan
• Joshua Cullip, Milton Keyes, Bucks, England
• Ben Scott, UK, Dementia at Seven story
• Leah Garfitt, UK
• Jesse Spenser, Melbourne, Australia
• Sawson Moubarak, Beirut, Lebanon

Children Lost To Niemann Pick Type C

• The Ara Parseghian Family’s Battle Against NPC
  • Video: Hail Mary’s Should Not Be Wasted On A Game
• Cathryn & Corynne Bouchard, Frisco, Texas, USA
  • Dallas Morning News Story on Bouchard’s Fight and Loss of their twins
• Kevin Eadie, Williamsburg, VA, USA
• Hunter Ozmer’s – “Will I Die?”
• Stacey Vorpahl
• Luke Liegghio, Canada
• Bryanna DeSouza
• Sam’s Story – Australia

Zero Cholesterol Butternut Squash Soup

ZERO Recipes Needed

Vegan Diet – No Animal Products We have eliminate virtually all cholesterol from Addi and Cassi’s diet, except for the occasional treats.  This includes meat, fish, chicken and all dairy products. While doctor’s don’t think diet plays a key role in NPC, we saw an immediate improvement in the twins’ stability within a week after starting a zero cholesterol diet.   While we are not advocating this diet, we have remained on this strict diet for over 1 year.  Today, Addi and Cassi eat soy, fruit, nuts or plant based foods but are taking Omega 3 fish oil which contains a small amount of cholesterol (10mls per day).   We understand DHA could be beneficial for progressive neurological diseases. Submit Recipes For Our Cookbook We decided we would create a section on our website and ask people to submit zero cholesterol  recipe ideas (or ideas for products that you love that don’t contain cholesterol).   Any creative idea is welcome.  We look forward to trying all your fabulous recipes.  Our dream is to compile a cookbook, which lists all the great zero cholesterol recipes submitted by people around the world with proceeds going to NPC research.   Help turn our dream into a reality by submitting a recipe today!  You can post ideas in the box below or email us.

Niemann Pick Type C Research Scientists

Mike Brown, Joe Goldstein, Rodney Infante
Brown and Goldstein Lab, University of Texas Southwestern, Department of Molecular Genetics

Daniel Ory
Washington University School of Medicine, St. Louis, Assistant Professor of Medicine, and Cell Biology and Physiology, Cardiovascular Division

Steve Walkley
Albert Einstein College of Medicine, Professor, Neuroscience

Lawrence S.B. Goldstein
Molecular Medicine Director, Howard Hughes Medical Institute, UC San Diego

Frances Platt
University of Oxford, Department of Pharmacology, Oxford, England

Christopher Austin
National Chemical Genomics Center, Director

Kyung-Sun Kang
Seoul National University, South Korea, Director, Stem Cell Program

Suzanne Pfeffer
Stanford University, Professor of Biochemistry

Synthia Mellon
UCSF, Developmental Regulation, Molecular Biology, and Novel Actions of Steroid/Neurosteroid Synthesis

John Dietschyand Benny Liu
University of Texas Southwestern Medical Center Department of Internal Medicine Division of Digestive and Liver Diseases

Frederick Maxfield
Cornell University, Professor and Chairman, Biochemistry

Matt Scott
Stanford University Bio-X Department

James Hildreth
McHerry Medical College, Director, Center for AIDS Health Disparities Research

Robert Erickson
University of Arizona, Professor of Genetics

Laura Liscum
Tufts University, Director of the Cellular and Molecular Physiology Department

Joyce Repa
University of Texas Southwestern Medical Center, Professor Physiology

Steve Sturley
Columbia University, Associate Professor of Clinical Nutrition

Charles Vite
University of Pennsylvania, Assistant Professor, Section of Neurology and Neurosurgery

 

Our Doctors

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Dr. Caroline Hastings
Oakland Children’s Hospital, Oakland, California
Renown Medical Center, Reno, Nevada

Dr. Caroline Hastings is currently overseeing Addi and Cassi’s compassionate use trial with hydroxy propel beta cyclodextrin (HPBCD) study that was approved by the FDA in March 2009. Dr. Hastings directs the inpatient hematology/oncology unit at Children’s Hospital Oakland and also manages the satellite hematology/oncology clinic at Renown Regional Medical Center in Reno, Nevada. We are very fortunate to have Dr. Hastings as part of our team as she has tremendous experience dealing not only with cancer cases but complex medical cases involving children.

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Dr. Mark C. Patterson
Mayo Clinic Department of Neurology
Rochester, Minnesota

Dr. Marc C. Patterson is one of the world’s foremost child neurologists with special expertise in neurometabolic and neurogenetic disorders, with a particular focus on Niemann-Pick Type C disease. Dr. Patterson travels around the world to meet with Niemann Pick Type C patients like Addi and Cassi. Here is a video of Dr. Patterson explaining the disease.

Dr. Patterson is consistently listed in “America’s Top Doctors” by his peers. You can read about his outstanding achievements by clicking on the links below:

• Dr. Marc Patterson’s professional background
• 1 year results of Miglustat – trade name “Zavesca”
• A Riddle Wrapped In Mystery – Niemann Pick Type C Disease:

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Dr. Forbes (Denny) Porter
National Institute of Child Health and Human Development
National Institutes of Health, Bethesda, Maryland

Dr. Forbes “Denny” Porter is one of the National Institutes of Health’s leading senior investigators in autosomal recessive cholesterol disorders including Niemann Pick Type C (NPC) and Smith-Lemli-Opitz syndrome (SLOS). Dr. Porter is currently conducting a natural history study at the NIH. In July 2009, Dr. Porter announced a therapeutic clinical trial with N-Acetyl Cysteine (NAC) to confirm the first plasma biomarkers in Niemann Pick Type C disease.

Addi and Cassi visited the National Institutes of Health in November 2007 and July 2008 to participate in the natural history study. This involved a week long trip to the NIH and multiple tests including 2 ½ hour special MRI scans, speech and swallowing testing, blood work-ups, a spinal tap and eyes exams. All tests are designed to look for biochemical markers in NPC children.  Our next trip is scheduled for October 2009.

Dr. Porter is currently looking to enroll additional Niemann Pick Type C patients into the N-Acetyl Cysteine trial. For more information about the study, contact Nicole Yanjanin at Dr. Porter’s office: 1-301-594-1765; nyanjanin@mail.nih.gov.

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Dr. Susan Sorensen
Private Practice
Reno, Nevada

Dr. Susan Sorensen, Addi and Cassi’s local pediatrician, was the first doctor to detect Addi and Cassi’s enlarged spleens (splenomegaly) through abdominal exams at 2 years of age. Dr. Sorensen has helped us run multiple tests to reach a diagnosis. If it was not for Dr. Sorensen pushing us to seek answers and encouraging us to see specialists outside of the Reno area, we may still not have a diagnosis.

• Be Your Baby’s Advocate:

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