Patient Advocacy Summit 2014: Patient-Centered Benefit-Risk Assessment, Why It Matters to You


Advocates at this year's 2014 Global Genes Patient Advocacy Summit will have an exclusive chance to learn how their voice can make a difference in the fight for faster cures. For critical patients and their families, the risk versus benefit of new treatments has been a simple decision. Although many are ready to take a chance on a new drugs, the complex course of procedure and legalities has … [more here...]

Mysterious Genetic Condition Complicates Life for Milo: Family Reaches Out


Our beautiful son, Milo, was born in December 2010. He has had a complicated life since then, with six surgeries and undiagnosed global developmental … [more here...]

“Boy Meets Girl,” New SMA Infographic Released by Gwendolyn Strong Foundation


The Gwendolyn Strong Foundation (theGSF) is a nonprofit organization dedicated to increasing global awareness of Spinal Muscular Atrophy (SMA), the #1 … [more here...]

Total Orphan Drug Presents: New Rare Syndromes


Novel form of congenital lipodystrophy and fatty liver disease due to biallellic loss-of-function mutations in PCYT1A in two unrelated patients The … [more here...]

Little Miss Hannah Foundation Seeks Donations for Care Bags


The Little Miss Hannah Foundation is going to be launching their new program in honor of what would have been Little Miss Hannah's 6th birthday (July … [more here...]

Ireland’s First Rare Disease Plan Published


Rare diseases, by their very nature, are one of the most challenging issues for any health service to deal with. A rare disease is defined in the EU … [more here...]

Drug Derived From Milk of Genetically Modified Rabbits to Treat Rare Swelling Disorder


The FDA has approved new drug, Ruconest, to treat people with Hereditary Angioedema. The new drug, developed in partnership by Salix Pharmaceutical … [more here...]