Double Trouble - Life With Identical Twins With Fatal Genetic Condition
One Mom's Journey Trying To Save The Lives of Her Identical Twins Who Have A Rare and Fatal Cholesterol Disease
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Feb 17, 12 07:00PMHere are the Rare Disease Day logos for 2012 from the Global Genes Project, the leading rare disease advocacy organization for the rare disease community. Since Rare Disease Day 2012 falls at the end of...
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Feb 06, 12 12:01PMI wonder if people truly understand what it means if their rare disease or disorder is on the RARE List™? Last week, the R.A.R.E. Project and Global Genes Project, leading patient advocacy organizations...
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Jan 15, 12 05:42PMWorld Rare Disease Day 2012 is coming up on February 29th. Consider these staggering statistics. Each year, 139 BILLION U.S. dollars are invested in medical research — much of it from tax payers...
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Dec 27, 11 06:05PMMy life grows more surreal by the day. Four years ago, I was told that my eight year old identical twins have a fatal genetic cholesterol disease called Niemann Pick Type C (the “Childhood Alzhe imer...
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Dec 22, 11 07:19PMIt’s great to see some representatives in Washington are finally starting to understand the real healthcare issue facing tens of millions of American’s afflicted with rare diseases. You ca n...
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Nov 15, 11 10:43PMThank you Dr. Francis Collins, Dr. Chris Austin, Dr. John McKew and others at the NIH who are all involved in driving the National Institutes of Health’s Therapeutics for Rare and Neglected Dise ases...
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Sep 14, 11 02:43PMLast year, after Dr. Caroline Hastings and I received the U.S. Food and Drug Administration orphan drug designation for Hydroxy-propyl-beta-cyclodextrin (HPBCD) for the treatment of Niemann Pick Type C...
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Aug 16, 11 02:48PMOver the past three years, many Niemann Pick Type C families as well as physicians treating NPC patients worldwide have contacted Dr. Caroline Hastings and I regarding Addi and Cassi’s treatment s...
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