National PKU Alliance


 

TRAIN Inventory Profile

Mission
Strategy/Approach
Research Portfolio
Financials
Key Accomplishments
Leadership
Ask Us About
 EXPAND ALL

Mission

The National PKU Alliance was created in 2008 by local groups of families all over the country who recognized the importance of a national agenda, fueled by their combined energy and dedication to find a cure, while working to improve the lives of individuals with phenylketonuria (PKU).

The alliance is a vital voice within, and on behalf of, the PKU community that serves a dual mission: improve the daily lives of those individuals and families affected by PKU, and accelerate the timeline for a cure by investing in peer-reviewed and targeted research.

Strategy/Approach

The NPKUA's four signature programs have the most significant returns for those who live with PKU: advocacy, education, support, and research.

Advocacy - NPKUA provides a unifying voice in the halls of Congress and in federal agencies to ensure PKU has a seat at the table in the national policy arena.

Education - NPKUA provides educational services for the full-life spectrum (initial diagnosis, school age, teen and adult support, and maternal health).

Support - NPKUA is the leader in the fight for insurance coverage for medical foods necessary to thrive as a person with PKU. And, while working on that long-term goal, NPKUA provides critical how-to Information to deal with the pressing issues with today's coverage hurdles.

Research - By consolidating the investments made for PKU research, our Scientific Advisory Board has focused resources on those research proposals that have the most promise both for improving the PKU community today and, ultimately, for a cure. This includes supporting the next generation of promising scientic researchers through its post-doctoral fellows program.

Research Portfolio

NPKUA's mission is to advance the science of PKU by funding the most promising peer-reviewed research that will lead to new therapy discoveries and a cure. Its primary goal is to have at least one new therapy in FDA clinical trials in the next five years. NPKUA wants to grow the quantity and quality of PKU research worldwide.

Currently, the organization funds novel research projects and fellowships. Initial funding can be renewed for a second year based on progress made.

Financials

Fiscal year 2014

  • Revenue: $990,209
  • Assets: $1,577,691
  • Grants: $570,045
  • Gifts Received: $950,027
  • Total Expenditures: $1,080,916

Key Accomplishments

NPKUA has invested more than $1.3 million in research grants aimed at developing new therapies and a cure. Many of these grants have allowed researchers to leverage additional funding to better understand the gaps in knowledge of PKU and accelerate projects related to a cure.

Accelerated a clinical trial by 3-4 years of hepatocyte liver transplantation in the U.S. as a possible cure.

Invested in research towards the creation of the first human variant of a genetically engineered probiotic that has now been purchased by a biotech firm.

Support three post-doctoral fellows to date to ensure that the most promising young scientists with an interest in PKU research are moved forward in their work faster.

Launched an international research challenge to develop a technology that would allow for in-home blood monitoring to better manage PKU.

Leadership

The NPKUA is governed by a 21-member board of directors, including 17 directors for its affiliate organizations across the country.

Its Scientific Advisory Board is comprised of 14 leading clinicians and researchers from around the world.

Staff

Ask Us About

  • Social media and communications
  • Strategic planning