Thank you for coming to the Family Fund Day 2015

Take Action! HR971

Action Alert
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

Open Act HR971

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:
Max G. Bronstein, MPP
Senior Director, Public & Government Relations
Phone: 415-884-0223
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

NPKUA Conference Survey

The survey link has been corrected, please accept our apologies!

Just a quick reminder…

One week to provide us with your much needed feedback!
If you haven’t already, please help us plan the 2016 conference by taking a brief survey on what you would like to experience at the next National PKU Alliance in Indianapolis, IN. The survey will be closed at 11:00 pm CST on Wednesday, April 15, 2015.

Take The Survey

Thank you for your participation! We truly value your feedback!


Christine Brown
Executive Director
National PKU Alliance
National PKU Alliance, Inc. | P.O. Box 501 | Tomahawk, WI 54487 |

Lobby Congress

Join us in May as we lobby on Capitol Hill!

We are pleased to invite you to lobby with us on May 18 and 19, 2015 for the Medical Foods Equity Act (MFEA) in Washington, D.C.! The MFEA would require all federal programs to cover the cost of medical foods (both formula and food modified to be low in protein) for children and adults with PKU and other inborn errors of metabolism.

The lobbying will take place on both Monday, May 18 and Tuesday, May 19. Volunteers will be paired with seasoned advocates for the lobbying appointments. If you can’t make it to D.C. in May, you can still help us by securing appointments for the NPKUA with your member of Congress. If you are interested in joining our efforts in D.C. or making an appointment for the NPKUA volunteers, please contact Amy Oliver at for more information and materials.

Information is also available on the NPKUA website – click here.

Thank you for your help in advocating for the MFEA! Together we can make our voices heard!

Thank you,
The National PKU Alliance