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

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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

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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

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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

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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

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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

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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

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