ZERO Recipes Needed
November 16, 2007 by Daddy
Filed under Zero Cholesterol Recipes
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
November 16, 2007 by Daddy
Filed under NPC Researchers
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
CureCaps Volunteer List
November 15, 2007 by Chris Hempel
Filed under Current Volunteers
Below is a list of our amazing CureCaps volunteers. Please join our cause today by emailing us.
California
Betty O’Connor, South San Francisco
June Heise, South San Francisco
Marlena Kien, Chester
Vi Kaupanger, Westwood
Peggy Van Wie, Auburn
Diane Crew, Elk Grove
Joyce Eriksson, Sacramento
Katarina Grabowsky, Sacramento
Sandra Neff, San Carlos
Judy Stogner, San Carlos
Lenore Sweet, Clayton
Eireann Schulenburg, Mountain View
Brigida Cortese, Montrose
Connie Christodulis, Los Gatos, constance_christodulis@yahoo.com
Janet Wasserman: wasserman_janet@yahoo.com
Shirley Mauricio, shirleym@1185design.com
Lisa Scott, lisaonvacation@hotmail.com
Sherif & Mereema Marmash, ray@arxnetworks.com
Colorado
Cathy Stevens, Greenwood Village
Massachusetts
Carol Chapman, Wakefield: cchaps225@verizon.net
Minnesota
Kathy Chapdelaine: kchapdelaine@carlsonwagonlit.com
Nebraska
Carol Eash, Omaha, vickithomas@tconl.com
New York
Barbara Danner, Rensselaer
Margaret Cipperley, Wynant Still
Nevada
Elaine Walker,Reno, elaine@eawalker.com
Linda Kelley: llkylemore@charter.net
Diane Shulman: brickshus@aol.com
Brooke Graham, brooke94509@yahoo.com
Atie Blinn, Reno, 775-853-9208
Gert Perez, Reno
Appelonia Ornelas, Reno
Debbie Dezsi, Reno
Liz Terry, Sparks
Jennifer Dolder, Reno, mjdolder@aol.com
Margaret Clark
Tennessee:
Abe’s Garden at Park Manor Retirement Community, mwomack@parkmanorapts.com
Texas
Marty Hughes: skymart46@hotmail.com
Virginia
Eric Childress, Virginia Beach, kelticshadow@aol.com
Wyoming
Saratoga Prayer Knitters
Marvin Worden
Barbara Johnson
Thank You Blue Cross Insurance!
November 11, 2007 by Chris Hempel
Filed under Insurance
Today is our first post in our blog and it’s a very significant day in our lives. We want to thank Anthem Blue Cross Insurance Company from the bottom of our hearts for giving Addi and Cassi a chance at life. Blue Cross has approved Zavesca for both girls and we plan to start the girls on it immediately. We have heard it doesn’t taste very good but we’ll manage that.
Blue Cross has been the most amazing insurance company since Addi and Cassi were born and we’re are going to beat Niemann Pick Type C disease with their help. The promising research announced by Brown and Goldstein labs shows what a central role Niemann Pick Type C plays in cholesterol metabolism.
On the other hand, we want the world to know how terribly disappointed we are in Actelion Corporation. Actelion, a Swiss based pharma company, does not have a “compassionate use” program established within their company. Compassionate use is a term used in the United States for “a method of providing experimental therapeutics prior to final FDA approval for use in humans. This procedure is used with very sick individuals who have no other treatment options. Often, case-by-case approval must be obtained from the FDA for “compassionate use” of a drug or therapy.
Actelion makes millions and millions of dollars each year (total net revenues of $946.4 million, up 38 percent since 2006) but DENIED our request for the only drug that could possibly help slow down Niemann Pick Type C. Do you think they could have considered splitting the cost with Blue Cross or could start a compassionate use program in order to support us and other kids suffering from Niemann Pick Type C? It’s all about the money. We are very fortunate that Blue Cross has compassion for children like Addi and Cassi and is supporting us in our fight against NPC.
Our Doctors
November 11, 2007 by Daddy
Filed under Our Doctors
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.
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:
Dr. Forbes (Denny) PorterNational 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.
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.
