1. 2nd International Homocystinurias Patient Expert Meeting
04 September 2017
Margie McGlynn
President, HCU Network America

2. Who is HCU Network America?
The Mission of HCU Network America is to:
Inform and provide resources for patients and families
Create connections across patients and families
Influence state and federal policy
Support advancement of diagnosis and treatment for HCU and related disorders.

3. Our Path to Forming HCUNAm:
First national Patient/Family Meeting held in Colorado in 2011
Relationships established and connections grew through Facebook
Business Plan for Patient Advocacy Group developed in 2015
Connected with HCU Network Australia through Facebook and meeting in Prague March 2016
Incorporated June 2016 and non-profit approved October 18, 2016

4. Our Path to Forming HCUNAm:
We formed our board in mid-2016
Kristin Rapp, Vice - President
Margie McGlynn, President
Danae Bartke
CFO & Secretary

5. Our Path to Forming HCUNAm:
We expanded our board and advisors in 2017 –
Kimberly Chapman
Children’s National
Washington D.C.
Harvey Levy
Boston Children’s
James Weisfeld-Adams
Children’s Hospital
of Colorado
Can Ficicioglu
Children’s Hospital of Philadelphia

6. Accomplishments
Website Built –

7. Accomplishments Resources available on Website
Description of Homocystinurias
Information for newly diagnosed
Patient stories
Brochure on HCU NA for patients and families
HCU Treatment Guidelines
“Find a Clinic” – listing by state
Formula and Medical Food Vendors
Financial assistance programs
Videos
Information on available clinical trials

8. Accomplishments Fund Raising Inaugural Campaign via Facebook 31 Donors
$19,195 + donor match!
Corporate Donors Being Solicited for:
-Advocacy Plan
-Support Clinic Outreach
-New patient toolkit
-Develop other educational materials,
-Webinar Support
-Newborn Screening
-2018 Patient/Family Meeting
-Investigate the current coverage and reimbursement landscape and change it!

9. Executive Director Hired
Webinar on Guidelines Held
March 1, 2017

10. Accomplishments Communication with community
Facebook page: HCU Network America
You Tube Channel: HCU Network America
Monthly Newsletters
Community Calls

11. Things to Come New Patient Toolkit
Expanded mailing list: patients, clinics & healthcare providers
Advocacy Plan
Webinar on Cobalamin Methylation disorders
Improve New Born Screening and Insurance Reimbursement
US Patient/Family Homocystinuria Conference
April 21-22, 2018
Boston, Massachusetts Suburbs

12. HCU – We’ve Come a Long Way…
1962: Homocystinuria first discovered!
1964: Low Met diet known to reduce levels and symptoms
1964: Doctors prescribed blend of amino acids to combine with low Met diet
1968: Folic Acid found to help manage HCU
1983: B6, B12 and folic acid were found to better treat high hcy levels
1985: Betaine found to help reduce hcy levels
1994: Cystadane (Betaine) approved for market in US
2014: HCU Network Australia founded
2016: HCU Network America founded
2018: Human trials for enzyme replacement therapy start, research for other new therapies progresses
But we need to keep working until a cure is found for all!