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 Michaux Family Foundation for PKU



Amy Oliver, Vice President Intermountain PKU and Allied Disorders



John LaPrad, Treasurer Michigan PKU and Allied Disorders



Kelly McDonald, Secretary North Texas PKU Association



Olaf Bodamer, MD, PhD



Heather Bomar, Tennessee PKU Foundation



Mary Brown



Rhonda Connolly, Children’s PKU Network



Mike Franklin, Indiana PKU and Allied Disorders Association



Jeb Haber



Virginia Harrison, MACPAD



Steve Hunt, Georgia PKU Connect



Ruthie Jager, PKU Organization of Illinois



Neil James



Stacey Kanihan, Minnesota PKU Foundation



Cait Kerr, Maryland Alliance of PKU Families



Melissa Perez, Florida PKU Foundation



Diane Pytel, California Coalition for PKU and Allied Disorders



Hollon Stevens, PKU Pursuits, Inc.



Ashley Sullivan, NECPAD



Mary Watkin , Iowa PKU Foundation

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.

• MEDICAL FOODS EQUITY ACT
• NEW MEDICAL AND DIETARY

GUIDELINES

• VOICE OF PKU AT THE NATIONAL

LEVEL

Advocacy

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

• GUIDE FOR NEW PARENTS
• WEBSITES
• CONFERENCES

Education

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

• INSURANCE COACHES PROGRAM
• MATERNAL PKU MENTORING

PROGRAM

• EMERGENCY FOOD ASSISTANCE

TO PREGNANT WOMEN

Support

Insurance Coaches Program

 Raenette Franco, Compassion Works  NPKUA Volunteers  Launched in February  Assistance to adults and families facing denials for

medical foods

Emergency Assistance Mentoring Program

 43 pregnant PKU

women in the last 20 months

 Cambrooke

Therapeutics and Nutricia

MATERNAL PKU

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

Research

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!