Welcome! Thank You Conference Sponsors Summit BioMarin Platinum - PowerPoint PPT Presentation

Welcome!

Thank You Conference Sponsors Summit BioMarin Platinum Hilton Salt Lake City Center Gold Vitaflo Nutricia Mead Johnson

Thank You Conference Sponsors Silver PKU Perspectives Cambrooke Therapeutics Bronze Cytonet Abbott Nutrition Kids Zone The Fletcher Bay Foundation

International Scientific Exchange Sponsors National Institute of Child Health & Human Development The Michaux Family Foundation for PKU Faiella Family and Comedy for Cure

Conference Committee  Amy Oliver, Chair  Jon Baker  Dr. Lorenzo Botto  Mary Brown  Sharon Ernst  Heidi Maxfield  Dr. Nicola Longo  Stacey Nelson  Heather Page  Kourtney Post  Sara Tye  Krista Viau

NPKUA Board of Directors Dick Michaux, President  Steve Hunt, Georgia PKU Connect  Michaux Family Foundation for PKU Ruthie Jager, PKU Organization of Illinois  Amy Oliver, Vice President  Intermountain PKU and Allied Disorders Neil James  John LaPrad, Treasurer  Michigan PKU and Allied Disorders Stacey Kanihan, Minnesota PKU Foundation  Kelly McDonald, Secretary  Cait Kerr, Maryland Alliance of PKU Families  North Texas PKU Association Olaf Bodamer, MD, PhD  Melissa Perez, Florida PKU Foundation  Heather Bomar, Tennessee PKU Foundation  Diane Pytel, California Coalition for PKU and Allied  Disorders Mary Brown  Rhonda Connolly, Children’s PKU Network Hollon Stevens, PKU Pursuits, Inc.   Mike Franklin, Indiana PKU and Allied Disorders  Ashley Sullivan, NECPAD  Association Jeb Haber Mary Watkin , Iowa PKU Foundation   Virginia Harrison, MACPAD 

Staff and Other Volunteers  Christine Brown  Katrina Swenson  Nick Bello  Ginny Michaux  Dr. Cary Harding  Desiree White  Kevin Brown  Karen Casey  Karen Amorde-Spalding  Belkys Prado

Who We Are Attendees  494 people  127 with PKU  39 states  9 countries

Housekeeping Restrooms  Outside the main doors of the ballroom Complimentary Internet in Meeting Spaces  Network: Hilton Meetings  Passcode: SLCCC14 Photo Booth  Listed on page 6  Today at 11:15 am and 4pm  Saturday at 5pm Lunch Today  Please clear room after general session ends so staff has enough time to reset for lunch

To improve the lives of individuals and families with PKU and pursue a cure.

Signature Programs advocacy, education, support and research The NPKUA is working to • help change the future of PKU – how it will be known, how it will be experienced and how it will be cured. By bringing the collective • experience of the PKU community to the table, the NPKUA is helping to shape the future of PKU.

Advocacy • MEDICAL FOODS EQUITY ACT • NEW MEDICAL AND DIETARY GUIDELINES • VOICE OF PKU AT THE NATIONAL LEVEL

HR 3665: The Medical Foods Equity Act • Addresses coverage of medical foods for life in federal programs • 20 co-sponsors • Covers 29 IEMs that are diagnosed at newborn screening or clinically (often during the first year of life)

How You Can Help  Info in Registration Packet  Schedule an In-District Visit  Congress in Recess: August 4- September 5

New Medical and Dietary Guidelines for PKU • Office of Personnel Management • CMS – Center for Medicaid and Medicare • Appeals

Voice at the National Level • FDA – Neurological Manifestations of Inborn Errors of Metabolism • National Institutes of Health • Secretary’s Advisory Council on Heritable Disorders in Newborns and Children

Education • GUIDE FOR NEW PARENTS • WEBSITES • CONFERENCES

Guide for New Parents  Based on our popular My PKU Binder  Distributed via metabolic centers across the country  First few years of life

Website  Clinic Locator  Cooking and Food  PKU Camps  Resources for School  Insurance  Legislation  Research Pipeline  Other Research

Teen and Adult Website

Conferences  Latest in PKU Research  Funded NPKUA Research  Daily Management  Networking  Teen and Adult Dinner  Adult Breakout Sessions  Teen Breakout Sessions  Kids Zone

Support • INSURANCE COACHES PROGRAM • MATERNAL PKU MENTORING PROGRAM • EMERGENCY FOOD ASSISTANCE TO PREGNANT WOMEN

Insurance Coaches Program  Raenette Franco, Compassion Works  NPKUA Volunteers  Launched in February  Assistance to adults and families facing denials for medical foods

MATERNAL PKU Emergency Assistance Mentoring Program  43 pregnant PKU women in the last 20 months  Cambrooke Therapeutics and Nutricia

Research F U N D E D R E S E A R C H H O M E P H E M O N I T O R S Y S T E M H O W Y O U C A N H E L P

NPKUA Funded Research  Since 2010, the NPKUA has awarded 18 research grants and 4 fellowships to improve treatment for PKU and find a cure  $1,037,000  Investing in targeted research moves the science of PKU and new treatments forward: - PEG-PAL - GMP and bone health - Liver cell transplantation

Home Phe Monitor • Raised more than $800k • Issued a Research Challenge to the International Scientific Community • Phases to Minimize Risk • Goal: Working prototype ready for commercialization

Raising Funds For Research – A New Initiative  Tuxes for Tia gala - $1.1 million for research  Launch of a branded fundraising gala series  Raise funds for research and support programs  Creation of an online gala toolkit via a web portal  Breakout session on Saturday at 1:30 pm – Creating Awareness and Events in Your Community

Raising Funds for Research

Donate to PKU Research  Send a Text to 41444  Text the word PKU  Followed by GIFT AMOUNT  Followed by YOUR NAME Help us raise $5,000 during the conference to accelerate the timeline for new treatment options and find a cure for PKU. Together, we can Reach New Heights!

Thank You!