Pfizer Announces FDA Approval of ELELYSO™ for Type 1 Gaucher Disease and Personal Support Program For Patients
On May 1, 2012, Pfizer announced the FDA approval of ELELYSO™ (taliglucerase alfa), an enzyme replacement therapy (ERT) for adults diagnosed with a rare genetic disorder called type 1 Gaucher disease. ELELYSO™, the first FDA-approved plant cell-based ERT for Gaucher disease, is also recognized for being acquired through a genetically engineered manufacturing system. This news [...]
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 [...]
Charity Tuesday: Courage to Make a Difference
It’s Charity Tuesday and we want to give a shout-out to a new charity called “Courage to Make a Difference” founded by Chris Dutcher. Chris has Hunter Syndrome (MPS II) and wants to spread awareness for all MPS diseases. To learn more, check out the great video below that Chris recently made. (The video was [...]
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 [...]
Brad Paisley Lends Voice to New Song "Hard Life" to benefit A-T Children's Project
Brad Paisley sings new song written by Joe Kindregan, A-T patient BRAD PAISLEY LENDS VOICE TO NEW SONG “HARD LIFE” Written By Joe Kindregan To Benefit A-T Children’s Project NASHVILLE, Tenn. (Dec. 12, 2011) Brad Paisley has just recorded a new song, “Hard Life,” available for purchase here http://links.causes.com/s/cly6Me and on all digital music outlets. The song [...]
HAPPY THANKSGIVING – from all of us at RARE Project
Wishing you and your families the joy of the season ~ celebrating with your loved ones. We are grateful for your involvement in this important movement, coming together, to support one another. ~ Warmest Wishes from all of us at the RARE Project. Nicole, Dean, Jason, Amy, Christie, Jen, Lisa, Meme ~ Recommend on Facebook [...]
50/50 and Neurofibrosarcoma
50/50, a film starring Seth Rogen and Joseph Gordon-Levitt based on the true story of Will Reiser who is a survivor from a battle against a rare cancer of the spine. // The Children’s Tumor Foundation seeks to improve the health and well being of individuals and families affected by the neurofibromatoses (NF).
New York Times Opinion – NOTES FROM A DRAGON MOM!
Dear Emily, So many of our community members sent me this story (ORIGINALLY PRINTED IN NY TIMES). Without question this story needed to be shared again and again. Emily, you are a part of a community of millions of Dragon Parents – incredible, strong, compassionate, tenacious, loving. . . I could go on. You, [...]
RARE DISEASE REGISTRIES 101
Disease Registries 101…is that when two diseases get married and pick out house wares? The term registry conjures different meanings to different groups. Couples use a registry when marrying. Hotels have registries of guests. And so do hospitals. There are also donor and recipient registries for hearts, kidneys, bone marrow transplants, etc. The thing these [...]