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 [...]
The Grassroots Movement Behind Ultra and Fast: Join the Movement!
A Guest Blog by Julia Jenkins: Director of Government Relations, EveryLife Foundation The Grassroots Movement Behind ULTRA-FAST & How you can join the Movement! We are very excited to share the news that rare disease Accelerated Approval language made it into the House draft PDUFA bill. The language that incorporates the goals of [...]
ULTRA Orphan Act – Fast Track Treatments for Ultra RARE Diseases
ULTRA Act UPDATE and REQUEST – Julia Jenkins, Everylife Foundation Congress is planning to introduce the Ultra-orphan Life-saving Treatments Act of 2012 or ULTRA Act to promote the discovery and development of safe and effective drugs and biologics to treat ultra-rare diseases. Your help is needed to ensure this legislation is successful! The ULTRA Act [...]
EVERYLIFE FOUNDATION BLOG – FDA Commissioner Appoints new leadership
The Right Person for the Job: FDA Commissioner Hamburg Appoints New Leadership As we continue to advocate for increased funding and improved specialized review divisions at the FDA, we are encouraged by the recent positive action coming from the Commissioner’s Office. Commissioner Hamburg has reorganized the top level of the Agency. We applaud her decision [...]
RARE Art contest deadline extended! Enter today!
The second annual RareArtist.org art contest is taking place now. What was so compelling last year was to see all the amazing art that was submitted from around the world. Our hats off to the Kakkis Everylife foundation for hosting this annual event. The artist and their work are very inspirational, this is important in [...]
Announcing the Launch of RareArtist.org an Online Gallery for Artists Affected by Rare Diseases
Announcing the Launch of RareArtist.org an Online Gallery for Artists Affected by Rare Diseases Art is the expressive medium that we all use when capturing the trials and triumphs of our human existence. For rare disease patients, life’s meaning takes on the greater significance that comes with struggling day to day with a devastating disease [...]
Organ Donors, Thalassemia, and Appropriations
An art sale benefits the New York Organ Donor Network; the Cooley’s Anemia Foundation battles on against a fatal genetic blood disorder; and the Senate supports orphan drug development and the treatment of rare diseases. Also, MarbleRoad is planning a big event for Saturday, October 9, 2010 in Washington, DC – please mark your calendars!
MarbleRoad Comes to the CRDN Blog
An introduction to Howard Liebers of MarbleRoad, who will be blogging weekly on the CRDN Advocacy/Policy channel.
Blog for Rare, a success and follow-up!
Feedback so far from Blog for Rare (I can’t resist a kind word): Thanks for the wonderful opportunity – Love this! (Tanya Lachance) Looks great, thanks Catherine! (Yolaine Dupont) Thanks! This has been on my To Do list for 4 days. ‘Glad I got it DONE! (Hug Your Kids Today) (Elizabeth Joshi commented on your [...]