Two-time Olympic silver medal winner: A champion for Hope
Adam Nelson Champions Hope Written by: Alan Abrahamson Through the eyes of two families affected by a rare disease, Alan Abrahamson, a seasoned writer for 3 Wire Sports, brings to us an amazing story of hope for Sanfilippo Syndrome and Niemann Pick Type C. In his writings, Alan delivers a realistic view of living [...]
The Global Genes Project Aims to Battle Rare Disease Through the 'Vote4Hope' Campaign and Pepsi Refresh Competition
The Global Genes Project Aims to Battle Rare Disease Through The ‘Vote4Hope’ Campaign and Pepsi Refresh Competition Over 15 Million Children in America Are Estimated To Suffer From 7000 Unique Rare Diseases; Children Unite As ‘Ambassadors of Hope’ To Win $250K Pepsi Grant For The Global Genes Fund DANA POINT, CA – September 1, 2010 [...]
Children's Rare Disease Network Partners With Medpedia.com To Create Rarespace
Online Knowledge Share Platform to Provide Valuable Information to Rare Disease Community Dana Point, Calif. (June 29, 2010) – Every parent needs a supportive, collaborative network full of information where medical professionals, researchers, patients, parents, advocates and the general public share knowledge about the rare childhood diseases that affect 22.5 million American families. RareSpace is [...]
R.A.R.E. Project's, Jonathan Jacoby to present at FDA Hearing on Rare Disease
On Tuesday, June 29, 2010, and Wednesday, June 30, 2010, FDA’s Office of Orphan Products Development will host a two-day public hearing and Webcast on the Development of Articles for Rare Diseases. This public hearing is intended to gain from health care providers, academia, industry, patients, and other interested persons their perspectives on various aspects [...]
Update: Orphan Drug Application (hydroxy propel beta cyclodextrin) by Chris Hempel
From Chris Hempel (Addi & Cassi Fund). Drum roll, please! Dr. Caroline Hastings at Children’s Hospital Oakland and Research Center received a call on Friday afternoon from the U.S. Food and Drug Administration regarding the orphan drug submission we made at the end of February. While we have not received the official letter by mail [...]
California Health Care Budget Crisis Threatens Life of Child Dying From Rare Disease
LOS ANGELES, California (CNN) — Anthony and Lisa Leoni have little time to worry about whether California’s budget crisis will affect their daughter’s life-sustaining care. A steady stream of nurses, caregivers and therapists visit 12-year-old Jessica at home around the clock. Jessica suffers from a rare and fatal disease called Niemann Pick Type C. A [...]
A Mom Brokers Treatment For Her Twins' Rare and Fatal Illness
Bucking Scientific Convention, Ms. Hempel Gets Researchers From Different Fields to Share Data on Potential Therapy By AMY DOCKSER MARCUS – April 3, 2009 F rom the moment her twin daughters, Addison and Cassidy, were diagnosed with a fatal genetic disease in October 2007, Chris Hempel has been searching for a drug that might save [...]
Collaboration is key in rare disease research!
KTVU-TV 2, the leading evening news in the San Francisco Bay Area, today reported on Addi and Cassi Hempel, identical twins affected by a rare disease called Niemann-Pick Type C (NPC). NPC is fatal and degenerative cholesterol disease that is often called the “childhood Alzheimer’s.” What is most interesting about this story is that Dr. [...]