The Power of Rare Disease Patients
A Guest Blog Post By Wendy White, Founder & President of Siren Interactive Thanks to the Pew Internet Foundation, we have a lot of data about ePatients. These empowered, engaged and educated patients (and families) are helping to bring about a transformation of healthcare in this country. Pew’s Susannah Fox has named them “healthcare superheroes.” Through [...]
Wall Street Journal Reports – Do It Yourself Drug Development, With Some Help!
The effort by two families to buy and develop a drug that holds promise in treating Duchenne muscular dystrophy — described today in the WSJ — is the result of an innovative new model set up to support the burgeoning phenomenon of do-it-yourself drug development. Before the Seckler and Wicka families bought halofuginone, a drug that [...]
Life Technologies makes a 'Big Hit' with rare disease patients and their families!
Life Technologies host rare disease families at the San Diego Padres Season Opener against the San Francisco Giants! What a great evening had by all who attended this exciting event hosted by Life Technologies. Rare disease families in the southern California area were treated to an afternoon of baseball in the Life Technologies Stadium Suite, [...]
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 [...]
Pepsi Refresh Ideas that Help Kids
We are crowdsourcing ideas from the Pepsi Refresh ideas campaign. If you know of a pending idea that benefits kids with rare disease, please post the url on the wall of the Children’s Rare Disease Network Facebook page so that we can add it to the Pepsi Refresh tab. Vote this link, cut and paste [...]
Searching for a Cure – Patient Perspective Podcast #1
In this first in a series of podcasts focused on rare disease patient advocates, we speak to Debra Miller, president and founder of Cure Duchenne. When Miller’s five-year-old son was diagnosed with Duchenne Muscular Dystrophy, she faced grim news. The progressive muscle loss caused by the disease typically leaves these children wheelchair-bound by age 10 [...]
CureDuchenne Hopes FDA Will Accomodate New Class Of Muscular Dystrophy Drugs Upon Proof Of Safety
Seven years ago, we were a happy family with mom, dad and a beautiful 5 year old boy named Hawken. We had noticed our son was unable to keep up in his first soccer team but we were assured by his pediatrician he was just a “late bloomer.” After insisting on some testing, we got [...]