Sempra Energy and Kids Helping Kids Volunteers Supporting Global Genes Project

Kyle and Jackson Watkins, with Lynn Watkins and Lisa Hubbard of Sempra Energy Supporting Global Genes Project

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 […]

CNN Health – Mom claims in viral blog that disabled child denied transplant

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(CNN) — Can a patient be rejected for a kidney transplant based on a developmental disability? A New Jersey mother alleges in her blog that this happened to her 3-year-old daughter, and it has sparked an Internet uproar. Whether a person with a mental disability […]

Rare Stories of Hope and Inspiration – Submit Yours Today

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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. […]

In A Parents Words – Why RARE Disease Needs ULTRA and FAST Act!

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Roy and Reed Zeighami, couldn’t have articulated it better – why the RARE Disease community needs the ULTRA Act/Fast Act of 2012. Please join Roy in viewing his two minute video!  And. . . if you feel so compelled, take action!!! ULTRA Act of 2012 […]

Do It Yourself Drug Development, With Some Help! – Wall Street Journal Reports

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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 […]

Costs of Caring for Children with Rare Disease: Online Survey

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The daily routines of those who care for children with rare genetic diseases are challenging.  In his new book, “The Boy in the Moon,” Ian Brown describes his personal struggle to cope with his son’s diagnosis of cardiofaciocutaneous syndrome. Brown details how he learned to communicate […]

75 Days Until World Rare Disease Day 2012!

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It’s December 15th and that means only 75 days left until World Rare Disease Day on February 29th, 2012! Other staggering statistics: Estimated 350 million people worldwide affected by rare disease Only 5% of rare diseases have treatments; there are no cures Over 50% of […]