ACTION ALERT – Accelerated Approval to treatments for rare disease patients!
The RARE Project has been working with Rare Disease Legislative Advocates (RDLA) on legislative improvements that would help bring more drugs to rare disease patients more quickly. This legislation promotes the use a variety of scientific data to qualify a surrogate endpoint for use in a clinical trial, when the required historical clinical information on [...]
NIH Supports Global Genes Project and Jeans/Genes Awareness Efforts
Over the past two years, the Global Genes Project efforts have been highly visible at the NIH for the annual World Rare Disease Day events. In 2012, we presented Dr. Francis Collins and Dr. Steve Groft with a Rare Pictures of Hope™ photo book made by thousands of rare disease advocates. In 2011, Nicole Boice, [...]
RARE Patient Stories – ‘Meet Reed!’
Meet Reed! In January of 2011, we learned that our then three and a half year old son Reed has Sanfilippo Syndrome Type A, a rare and fatal Neurodegenerative disease. The disease is very rare, affecting about 1 in 70,000 births. To put that in perspective, according to NOAH, you are seven times more [...]
RARE Patient Stories – ‘Meet Connor!’
‘Meet Connor!’ Connor is a typical 5 year old boy, except, he has a life threatening condition. He loves to play with cars, hang out with his friends, and pretend to be Spiderman. But sometimes the fun times take a back seat when he is home sick with a cold because of his low immune [...]
Take Action: Tell Congress Rare Diseases Patients Deserve Treatments
Tomorrow is a day of celebration, but TODAY is a day of ACTION. More than 70 patients advocates are on the Hill today meeting with Members of Congress. Please join them in the Lobby Day by taking 10 minutes to call your Representatives and ask them to support efforts to spur lifesaving treatments for rare [...]
PATIENT STORIES OF HOPE AND INSPIRATION – ‘Meet Peyton!’
Peyton isn’t that much different than any other four year old if you ask her. She loves to color and dance and she is a ‘Yo Gabba Gabba’ fanatic. Luckily, she is still blissfully unaware of the stares directed her way when we go to the grocery store or the playground. Peyton was born with [...]
Charities Unite Worldwide to Fund Research into Reversibility of Brain Damage Caused By Sanfilippo Syndrome
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 type of therapy or treatment, and much of the early [...]
Sempra Energy and Kids Helping Kids Volunteers 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 children and families affected by rare disease. ”It has been [...]
GUEST BLOG: A special collaboration among the RARE Project, Grand Rounds, and The Afternoon Nap Society
Guest Blog by Sarah E. Kucharski of the Afternoon Nap Society A special collaboration among the RARE Project, Grand Rounds, and The Afternoon Nap Society will use social media to connect patients and healthcare providers while raising awareness of World Rare Disease Day (coming up on February 29). As a rare disease patient and healthcare [...]
ACTION ALERT – World RARE Disease Day at National Institute of Health
Rare Disease Day at NIH (RDD@NIH) On February 29, 2012, the National Institutes of Health (NIH) will celebrate the fifth annual Rare Disease Day with a day-long celebration and recognition of the various rare diseases research activities supported by the NIH Office of Rare Diseases Research, the NIH Clinical Center, other NIH Institutes and Centers; [...]