MarbleRoad: A Storybook Success
Helping To Raise Awareness MarbleRoad, a non-profit based in DC wanted to make a creative impact to raising awareness to families in need. Their mission: ….” to connect people who have complex illnesses with the resources they need to help them improve their lives.” And with the help of RAREProject and others, MarbleRoad accomplished their [...]
Submit Your Story to the Rare Children's Storybook Project by January 13
Has your family submitted to the “Rare Children’s Storybook Project” yet? Your story could win up to $750 to help with medical costs. The deadline has been extended to January 13. MarbleRoad who is managing this promotion was awarded an “Empowerment Grant” from Genzyme to hold an online contest within the rare disease community for [...]
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 [...]
A Rare Disease Pioneer Scores $45 Million for New Company
When Emil Kakkis was a researcher at Harbor UCLA developing what would become the first enzyme replacement therapy, he says pharmaceutical companies wouldn’t give him the time of day. The treatment was eventually developed and marketed as Aldurazyme by BioMarin and its partner Genzyme. Kakkis, who eventually became chief medical officer of BioMarin, has just raised $45 [...]
KIDS GET COMMUNITY SERVICE LESSON – Rare Disease Awareness in Las Vegas
Vanderburg students make bracelets, ribbons to recognize World Rare Disease Day By JEFF MOSIER VIEW STAFF WRITER, Green Valley Henderson VIEW JEFF MOSIER/VIEWRobert Ostrea, shown with his daughter Hannah and son Ethan, attend an event in Ethan?s fourth-grade classroom Feb. 28 at Vanderburg Elementary School, 2040 Desert Shadow Trail. The students worked to make bracelets [...]
World Rare Disease Day Efforts Underway from Global Genes Project
Global Genes Project volunteers working on multiple programs to build awareness and support World Rare Disease Day, Feb 28, 2011! Ever want to be part of something bigger? Ever want to really feel like people are helping champion your cause. Well. . . they are. The Global Genes Project was developed by the rare disease [...]
Rare Disease Drugs: A $1 Trillion Market?
Matthew Herper The Medicine Show; FORBES November 2010 Right now drugs for rare disorders are the sweet spot of the biotechnology industry. Treatments for illnesses that affect only hundreds of patients can cost $200,000 or more and have built Genzyme into one of biotech’s biggest companies and Alexion and Biomarin into two of its hottest [...]
Thank you Frances Perraudin – YES, It Is Time The World Pays Attention To Rare Disease!
Time Magazine and Frances Perraudin’s article, “Is It Time We Paid More Attention to Rare Diseases?” articulates the inherent challenges faced by the rare disease community and briefly introduces you to Carrie Ostrea and her daughter Hannah who suffers from a rare disease called Neuronopathic Gaucher’s disease. Yes, a global rare disease registry is critical [...]
Blog for Rare! Bloggers from around the world come together for rare disease awareness and support for World Rare Disease Day 2010 (Feb. 28). #blog4rare
Welcome to the first official “Blog for Rare.” We’re bringing people from across the world together this week in celebration of World Rare Disease Day 2010 (February 28). This SNiPs post is a mix of web links to specific blog posts and to bloggers who have a connection to rare disease (a blog roll [...]