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 [...]
World Orphan Drug Congress: Harmonization
Takeaways from the World Orphan Drug Congress R.A.R.E. Guest Blogger: Eileen O’Brien Director, Search & Innovation Siren Interactive The World Orphan Drug Congress, I attended in April consisted of three days jam-packed with presentations and networking. While the event focused on rare diseases and orphan drugs, the scope was still wide and addressed issues ranging from [...]
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 [...]
CHI Reports – How Biotech Companies are Shaping the Future in Rare Diseases
How Biotech Companies are Shaping the Future in Rare Diseases Biotech companies are navigating new trends in addressing rare diseases, conditions effecting 200,000 or fewer Americans. Jeremy Springhorn (Alexion Pharmaceuticals), Andrew Curtis (Pfizer), Marc Beer, (Aegerion Pharmaceuticals) and Susan Kahn (National Tay-Sachs & Allied Disease Association), shared their perspectives on the future of new treatments [...]
CHILDREN'S HOSPITAL BOSTON PARTNERS WITH PFIZER – FOCUS ON ORPHAN AND GENETIC DISEASES
Children’s partnership with Pfizer: A new way to speed therapeutic development by Nurjana Bachman on June 10, 2011 Over the past nine months, Pfizer has built collaborations with a number of premiere academic medical centers, including Children’s Hospital Boston. Wednesday marked the launch of the Boston branch of Pfizer’s Centers for Therapeutic Innovation (CTI), fostering [...]
PARENT DRIVEN RESEARCH – Innovations from the trenches!
A Push to spur more drugs for deadly rare diseases LAURAN NEERGAARD, AP Medical Writer Cassidy Hempel, 6, waves at hospital staff with the help of her mother Chris at the Children’s Hospital and Research Center in Oakland, Calif., Friday, March 18, 2011. Cassidy and twin sister Addison are receiving alternative treatment for Niemann Pick [...]
GLOBAL GENES PROJECT – NEWS AND UPDATES
Global Genes Project E-News It’s a Wrap – Rare Disease Day 2011. DANA POINT, Calif. – The 4th Annual World Rare Disease Day took place February 28, 2011 with a fanfare of activities worldwide! Sponsoring organizations like NORD and Eurordis engaged in more activities and awareness efforts, garnering increased attention and engagement than previous years. [...]
GLOBAL GENES PROJECT ANNOUNCES “WEAR THAT YOU CARE™” DENIM AWARENESS CAMPAIGN FOR RARE DISEASE DAY 2011
Statistics Show 95% of Rare Diseases Have No FDA Approved Drug Treatments; Only 352 New Drugs Developed and Approved For Tens of Millions of People Since 1983 DANA POINT, Calif. – January 26, 2011 – Monday, February 28, 2011, is the 4th annual Rare Disease Day — a day when people worldwide will show their [...]
World Rare Disease Day Efforts Underway from Global Genes Project
Global Genes Project volunteers working on multiple programs to build awareness and support World Rare Disease Day, Feb 28, 2011! Ever want to be part of something bigger? Ever want to really feel like people are helping champion your cause. Well. . . they are. The Global Genes Project was developed by the rare disease [...]
Kids Helping Kids – Denim Ribbon's being made to support World Rare Disease Day!
Over 50 kids and their families descended upon the RARE Offices to help make denim ribbons in preparation for World Rare Disease Day taking place this February 28, 2011! It was inspiring to see so many kids, that I am sure would have rather been playing with their new toys and hanging out with their [...]