Announcing RARE Project’s Newest Program
RARE Meetups and Local Leaders Program We are very excited to announce the roll-out of RARE’s new community support program “RARE Meetups and Local Leaders.” The very first RARE Meetup will be held in the South Orange County area (Southern California) on Thursday, May 31, 2012 from 10:00 a.m. to approximately 12:00 noon. All [...]
FOR IMMEDIATE RELEASE – HR 5651, the Food and Drug Administration Reform Act of 2012
FOR IMMEDIATE RELEASE House FDA User Fee Bill Will Unlock Lifesaving Treatments for Rare Diseases Rare Disease Community Applauds House Energy & Commerce Committee for User Fee Reauthorization Legislation that Spurs the Development of Treatments Thursday, May 10, 2012, WASHINGTON, DC—– Today the House Energy & Commerce Committee finished mark-up of HR 5651, the “Food and [...]
Place your Bets! Olympian Needs Your Support for MPS – Sanfilippo and RARE Project
Adam Nelson is the rarest of individuals. He is an Olympian. He is even rarer, as the man has won World Championship and Olympic medals in one of the most competitive events in our sport. Here is how it works: if Adam Nelson gets behind a cause, we support it. Do something for a higher [...]
PKU and the Adventures of Luke and his Incredible Family
Luke was born February 12, 2007, the seventh of our beautiful children. At four days old we received a phone call that Luke’s new born screening was showing an inborn error. So we were sent to Johns Hopkins that same day where we learned Luke had PKU, or phenylketonuria. Being the 7th of our children, [...]
CNN Health – Mom claims in viral blog that disabled child denied transplant
(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 qualifies for an organ transplant is a decision made on [...]
Improving Drugs and Biologics for Children – Washington DC Briefing
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ACTION ALERT: Costs of Caring for Children with Rare Disease Survey
Shivani B. Nazareth, RARE Blog contributor and a board-certified genetic counselor with Counsyl, Inc, is conducting an anonymous survey to better understand the financial cost of caring for a child with a rare genetic disease. By participating in this survey, you will contribute to a research paper that will quantify the financial burdens endured by parents of [...]
Submit your art & win an iPod Touch!
Calling all artists, kids, parents, friends & family! Share your art that captures the beauty, power, love, pain and courage of the rare disease community. Words from a past winner, we couldn’t have said it better: “ This is so much more than just a simple contest to me; this is about being a part [...]
Costs of Caring for Children with Rare Disease: Online Survey
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 with his son, who is unable to speak, and how [...]