A RARE DISEASE PATIENT PERSPECTIVE – Roy Zeighami
A Rare Disease Patient Perspective I would like to use this blog post to explain why the rare disease community is different, and why it deserves a different approach to the approval of treatment. This weekend I started reading Inside the FDA: the politics behind the food we eat and the drugs we take. In [...]
'R.A.R.E.' BOARD OF DIRECTORS AND ADVISORS ANNOUNCED
The RARE Project Announces Big Team Additions, Setting the Stage For Continued Success Thirty (30) million people in the US and 350 million people worldwide are affected by rare disease. Rare disease affects more children and their families then AIDS and CANCER combined worldwide. The RARE Project, developed by various rare disease leaders, is an [...]
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 [...]
When RARE is not so RARE!!!
When RARE is not so RARE! By Nicole Boice, Founder/President, RARE Project Did you know that there is a community of sick and disabled women, children, and men that is so large that it outnumbers all of those with cancer and AIDS combined worldwide? Scary to think about? Can you guess what it is? Staggering [...]
Wall Street Journal 'Health Blog' Reports – GETTING MORE DRUGS APPROVED FOR RARE DISEASES
By Amy Dockser Marcus A new paper offers a possible road map for getting more drugs approved to treat rare diseases. One key recommendation: improving access to the FDA’s accelerated approval pathway, originally developed to quickly get treatments to patients with life-threatening diseases. The paper, written by Brigitta Miyamoto and Emil Kakkis of the Kakkis [...]
A Rare Disease Pioneer Scores $45 Million for New Company
When Emil Kakkis was a researcher at Harbor UCLA developing what would become the first enzyme replacement therapy, he says pharmaceutical companies wouldn’t give him the time of day. The treatment was eventually developed and marketed as Aldurazyme by BioMarin and its partner Genzyme. Kakkis, who eventually became chief medical officer of BioMarin, has just raised $45 [...]
Hereditary Inclusion Body Myopathy (HIBM) HAS NEW CHAMPIONS AND REAL HOPE
Rocking It For Rare Disease! If anyone can do it, Dr. Emil Kakkis can. One of the rare disease communities scientific champions (also one of GQ Magazines recognized Rock Stars of Science), Dr. Kakkis comes at his science with a passion and ferver that moves mountains. How incredibly lucky is this community to have a [...]
RARE Artist – ART CONTEST WINNERS Announced
This year’s contest produced another amazing collection of beautiful artistic expression. We received many incredible and moving works of art in a variety of mediums, including paintings, drawings, quilts, photography, sculptures and glass work. Entries to this year’s contest grew by nearly 50% from our inaugural contest with our Board members reviewing nearly 200 original [...]
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 [...]
FDA and Rare Disease Drug Development, from FDA Week
June 29, 2010 // [FDA Week, http://insidehealthpolicy.com/] FDA is currently in the middle of a flurry of activity aimed at better focusing its regulation of rare disease treatments, but the agency disappointed some outside advocates by delaying the possibility of creating a special rare disease review division within FDA for at least a year. That [...]