Month: May 2015
Take Action! HR971
Take Action!
OPEN ACT & United Rare Disease Day
Please call Congress TODAY and ask them to include the OPEN ACT, HR 971 (Orphan Product Extensions Now, Accelerating Cures & Treatments) in the 21st Century Cures Legislation. The OPEN ACT has the potential to double the number of approved rare disease treatments available to patients. To date, 155 patient organizations support the OPEN ACT, including NORD, Global Genes, and the Genetic Alliance. By standing together we can ensure Congress helps rare disease patients.
Click here to take action: Stand up for Rare Disease Patients TODAY
Please share this alert widely and join our event on Facebook.
Why the OPEN ACT is important: Despite advances made possible by the Orphan Drug Act, 95 percent of the 7,000 rare diseases still have no approved treatments. Biopharmaceutical companies are not repurposing major market therapies to treat rare diseases because there is no incentive for them to do so.
The OPEN ACT is bipartisan legislation that creates an economic incentive for companies to repurpose drugs for rare diseases. The OPEN ACT could:
• Bring hundreds of treatments to rare disease patients
• Enable access to safe, effective and affordable treatments
• Spur biotech investment, innovation, and foster clinical research at universities while creating new jobs
Learn more at: http://curetheprocess.org/incentivize/
Sincerely,
Max G. Bronstein, MPP
Senior Director, Public & Government Relations
Phone: 415-884-0223
EVERYLIFE FOUNDATION FOR RARE DISEASES
Accelerating Biotech Innovation for Rare Disease Treatments
Take Action Now – ask your representative to co-sponsor the OPEN ACT HR 971 to help bring treatments to rare disease patients!
Where do your donations go?
It may also be helpful to know that since 2010, we have invested $1.3 million in research. Some of our initial successes with these funds raised primarily by local organizations have resulted in:
• Accelerating a clinical trial by 3-4 years of hepatocycte liver transplantation in the first U.S. patient as the result of funding two fellows at the University of Pittsburgh
• Research towards the creation of the first human variant of a genetically engineered probiotic at the University of North Texas has been purchased by a biotech firm
• Leveraging NIH funding for gene therapy at Oregon Science and Health University
• Leveraging studies in the mouse model to translational studies in human PKU on skeletal fragility and have resulted in several publications in peer reviewed scientific journals of University of WI
You can also access the full impact of funding document at http://www.npkua.org/Portals/0/PDFs/research/Impact_of_Funding_2014.pdf.
Event: Parent Cafe on June 6th
Our second event of the year is right around the corner! A no charge event in great location to get together with parents and children. Your kids can play together in a three story playhouse while you talk with other parents and caregivers! Register by clicking below. Registration closes May 30th!!
