Building awareness and communities to support and find treatments for Rare Diseases and Genetic Conditions
Sometimes, it pays to be secretive: Isis Pharmaceuticals received a $5 million milestone payment from GlaxoSmithKline (GSK) toward the development of a drug to treat an undisclosed rare and serious disease. Isis will develop the drug to Phase II proof-of-concept, at which time GSK has the option [...]
There are less than 30 days to World Rare Disease Day on February 29. We kick off our campaign this year as host of a Rare Disease Blog Hop. Over 40 bloggers united to blog today to raise awareness about this important day and on [...]
Charities Unite Worldwide to Fund Research into Reversibility of Brain Damage Caused By Sanfilippo Syndrome (RARE Project – Dana Point, CA) Rare Disease affects 350 million people worldwide, consisting of 7,000+ identified diseases and disorders. Currently less than 5% of all rare diseases have any [...]
Sempra Energy Foundation and Southern California rare disease advocates work to raise awareness and volunteer for the Global Genes Project, a RARE Project initiative. Family and friends gathered together to help make denim ribbons in support of the Global Genes Project’s efforts to support those [...]
Thank you to all those who have submitted stories of hope and inspiration! We’ve received many heartfelt stories from parents and patients of those affected with Moebius Syndrome, CPS (Carbamoyl Phosphate Synthetase), Dystonia, undiagnosed, Glut1 DS, Krabbe’s Disease, Charcot Marie Tooth Disorder and many more. [...]
The laws named the Best Pharmaceuticals for Children Act and Pediatric Research Equity Act, or BPCA/PREA, have resulted in over 400 drug labels. Be sure to check out the speakers at this event sponsored by BIO.
In December 2011, U.S. Representatives Cliff Stearns (R-FL) and Ed Towns (D-NY), two senior members of the Energy and Commerce Committee, introduced bipartisan legislation to help spur the development of treatments for very rare diseases. The Unlocking Lifesaving Treatments for Rare Diseases Act of 2012, [...]
