MLD Family Conference™ ... Suffolk, Virginia - 2017

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Registration Closed
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  Online registration for this conference closed on Monday, June 12. If you would still like to register we will try to accommodate you as best we can. Please contact us via email or at +1 503-656-4808.

LIVE WEBCAST: There will be plenty of space on the free live webcast, which will appear on the Conference Overview page during the conference.

*** We are sorry, due to planning and space constraints we cannot accommodate on-site registration or walk-in attendees. ***

2017 MLD Family Conference™ 2017 AGENDA

Sponsored by the MLD Foundation

July 14th–15th in Suffolk, Virginia

July 13, 2017– subject to change ... See last year's agenda here

Friday, July 14th

Friday, July 14th


Registration opens

Sea-esta Cove (Respite Room) opens

Light Continental Breakfast

Foyer - (outside Kilby)




Welcome & Personal Introductions



MLD, What’s in a Name? – Examining the biochemical roots of MLD and what it means to be a Lysosomal Disease/Leukodystrophy

Shire Study Summary - Diagnostics, Symptoms and Progression

facilitated by Brett Crawford Ph.D., BioMarin
Discussion - All

Magdalena Harrington PhD, Shire


Lunch (Friday Industry and academic experts photo)



Progress Toward a Treatment – hear and discuss the latest going ons in MLD research and learn the steps in the research process

Gene Therapy with Transplant – Milano

Discussion of other research and next steps

Francesca Fumigali MD, San Rafaelle SR-TIGET
Discussion - all


Ladies Meet-up and Mens Meet-up – A time for those new and for those more experienced on the MLD journey to come together in a small, caring, confidential environment

Academia & Industry Brainstorm – A brief brainstorm of priorities for future MLD research, clinical, and advocacy efforts

MLD Parents/Caregivers

Academia and Industry Attendees





Navigating the Medicaid Maze – Learn the ins and outs of Medicaid, who is eligible, what it provides, how to apply

Inez Loyd, Social Security Administration


Progress Toward a MLD Newborn Screen – Learn how the WA State NBS pilot is going and what are the next steps

Prof. Michael Gelb
, Univ. of Washington

Discussion - All


MLD & Rare Disease Advocacy – Explore the policy issues, learn how to make a difference that matters

Discussion - All


Family Time




Followed by Suffolk Community concert on the lawn or up in room 320



MLD After Hours ... You are invited to MLD After Hours after the concert winds down. Put the kids to bed, kick off your shoes, and come on up to join us for an informal time of sharing and conversation. Who knows, you might even find some surprises to pass the time!

MLD Central – Presidential Suite, room 320

Saturday, July 15th

8:00am – 9:30




Registration opens

Sea-esta Cove (Respite Room) opens




After the Diagnosis - Now What? The Role of Healthcare Advocates in Managing Patient Care – Hear how a Lysosomal Center approaches patient care, how a healthcare advocate can help, get tips on handling insurance denials

Stephanie Cagle MS, Emory University

Discussion - All





Putting the Educational Puzzle Together – Explore the educational resources available & how they can help you and your child

Gigi Durr, ESE Inclusion Specialist

Discussion - All


Group Photo

photo location TBD



Constant’s Wharf & Veranda


Butterfly Ceremony – A special remembrance of those who have earned their Butterfly Wings since our last time together and honoring all our MLD Angels

photo location TBD


Building the MLD Journey Toolbox - Possible Head to Toe Roadblocks – Explore and discuss issues that can come up along the MLD journey and possible solutions to have in your toolbox

- Maria Escolar MD, UPMC member of one of the MLD Boards
- Adeline Vanderver MD, CHOP member of one of the MLD Boards
- Sarah Chagdon MD, CHKD

Discussion - All





CBD Oil - Education & discussion – Explore the creation of Hailey’s Hope and learn about types of CBD Oil & dosing

Jason Cranford, Flowering Hope Foundation (via live web)

Discussion - all


Pulling It All Together ... Group Hug



MLD children ... Gather in Chuckatuck for formation of Celebration Parade



Parade and MLD Family Beach Party



MLD After Hours ... Put the kids to bed, kick off your shoes, and come on up to join us for an informal time of sharing and conversation.

MLD Central – Presidential Suite, room 320

tbd - to be determined

member of one of the MLD Boards Member of the MLD Foundation's Medical and Scientific Advisory Board (MSAB) or the MLD Foundation Board (fiduciary)

Sunday, July 16th

Sunday is an optional social day.
Hotel checkout is at noon

Thank you to our sponsors, service, & grant providers:

Believing For Bryleigh   Shire Pharmaceuticals   Honoring Olivia   Soulumination  
who through their sponsorships, grants, and support are making this conference much more affordable for the attending families.

We also wish to thank the individual families for their donations to the scholarship fund.

Industry and Academia Attendees

Name Institution Location Title/Specialty

Michael Babcock PhD

BioMarin Pharmaceutical

San Leandro, CA

Discovery Scientist & Biologist

Stephanie Cagle MS

Emory University

Atlanta, GA

Genetic Counselor

Randell Walton


Seattle, WA


Sarah L. Chagdon MD

Children’s Hospital of the King’s Daughters

Norfolk, VA

Pediatric Neurologist

Jason Cranford (via live web)

Flowering Hope Foundation

Boulder, CO

Founder and CBD Botany Specialist

Brett Crawford PhD

BioMarin Pharmaceutical

Novato, CA

Staff Scientist

Erika De Boever PhD

GSK - GlaxoSmithKline

Raleigh-Durham, NC

Sr. Director, Rare Diseases Clinical Development

Gigi Durr

Hernando County School District

Spring Hill, FL

ESE Inclusion Specialist

Maria Escolar MD *

Univ. of Pittsburgh Medical Center

Pittsburgh, PA

Developmental Pediatrician, Director - Program for the Study of Neurodevelopment in Rare Disorders (NDRD)

Francesca Fumagalli MD

San Raffaele Hospital, Telethon Institute for Gene Therapy (SR-TIGET)

Milano, Italy

Neurologist Consultant

Michael Gelb PhD

University of Washington

Seattle, WA

Professor of Chemistry and Biochemistry

Magdalena Harrington PhD

Shire Pharmaceuticals

Lexington, MA

Director, Health Economics and Outcomes

Mark Lenker

Shire Pharmaceuticals

Washington, DC

Director, US Government Relations and Public Policy

Inez Loyd

Social Security Administration

Norfolk, VA

Public Affairs Specialist

Joseph Phillips PhD


Calabasas, CA

Sr. VP & Head of Pharmaceutical Development

Andrew Shenker MD PhD

GSK - GlaxoSmithKline

Philadelphia, PA

VP, Metabolic Disease Area Head

Dean Suhr

MLD Foundation
Dad to Lindy and angel Darcee

West Linn, OR

President & co-Founder

Teryn Suhr

MLD Foundation
Mom to Lindy and angel Darcee

West Linn, OR

Executive Director & co-Founder

Paul Szabolcs MD

Univ. of Pittsburgh Medical Center

Pittsburgh, PA

Chief, Blood and Marrow Transplant & Cellular Therapy

Adeline Vanderver MD *

Children’s Hospital of Philadelphia

Philadelphia, PA

Pediatrician, Neurologist, Director of Leukodystrophy Center of Excellence

Margaret Wasilewski MD

Shire Pharmaceuticals

>Lexington, MA

Global Clinical Development Lead for Rare Disease Neuroscience


Duke University Medical Center

Durham, NC

PBMT Cellular Therapy Program

Contact Dean Suhr <> +1 503-656-4808, USA: 1-800-617-8387, or FAX +1 503-212-0159
This conference is organized by the MLD Foundation. Check with your tax advisor about the deductibility of any expenses associated with this conference.

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