Building awareness and communities to support and find treatments for Rare Diseases and Genetic Conditions
More than three million people in the United States suffer from epilepsy at an estimated cost of 15.5 billion annually. In the United States, more than 300,000 people with epilepsy are under the age of 14 and more than 500,000 are over the age of 65. [...]
The Williams Syndrome Changing Lives Foundation is launching a direct donation program called PROJECT 7. This program allows donors to sponsor an individual with Williams syndrome. Donations made to specific individuals will assist with their medical, developmental, educational, recreational and therapeutic needs. A hundred percent of [...]
In 2010, the Williams Syndrome Association designated the first full week in May as “Williams Syndrome Awareness Week.” In the years since, the number of awareness events for Williams Syndrome held annually has grown tremendously, and this year, Congressman Jim Moran will introduce a resolution [...]
Dear Warriors, Have the last five years of chronic stress and uncertainty been accompanied with hope? There are varying degrees of hope, and I will share what ours have been. We have had hopeful years. Hopeless days. We have hoped the ongoing nightmare would come to [...]
Just one moment… As a documentary filmmaker sometimes that’s all I’m looking for. One moment that defines the story, something I couldn’t have planned for in pre-production, something that would never make it to the shot list because I couldn’t have imagined it. And it’s [...]
My eldest, a girl aged eight, is very prim and proper. She likes getting 10 out of 10 in her spelling tests, finishing all her homework and getting certificates. She would be mortified to arrive late for school, and she refuses to break any rules ever. If [...]
A key theme of the World Orphan Drug Congress 2013 was the need to innovate when it comes to the rare disease space. Presenters shared how rare diseases necessitate a different approach, ranging from clinical trials to working with patients and the US Food and [...]
Nimbus Discovery splashed onto the scene in 2011 with a big-name investor group that included Bill Gates and a plan to discover drugs with the help of computer software. On Tuesday, that idea turned into a potentially big partnership with Shire to help conquer currently untouchable rare [...]
LYSOGENE announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its lead gene therapy product SAF-301 for the treatment of Sanfilippo type A syndrome (also named mucopolysaccharidosis type IIIA), a rare, genetic and life-threatening disease affecting children (http://www.accessdata.fda.gov). [...]
