9:00am

Registration opens

Sea-esta Cove (Respite Room) opens

Light Continental Breakfast

Foyer - (outside Kilby)

9:30

Welcome & Personal Introductions

Everyone

10:15

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

12:15

Lunch (Friday Industry and academic experts photo)

Meade

1:15

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

2:00

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

3:00

Break

3:15

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

3:45

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

4:15

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

Discussion - All

5:00

Family Time

6:00

Dinner

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

Meade

9:00-12:00

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

8:00am – 9:30

Breakfast

Meade

9:00

Registration opens

Sea-esta Cove (Respite Room) opens

Foyer

Chuckatuck

9:30

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

10:30

Break

Foyer

10:45

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

12:00

Group Photo

photo location TBD

12:30

Lunch

Constant’s Wharf & Veranda

1:45

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

2:15

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

Panelists
- Maria Escolar MD, UPMC
- Adeline Vanderver MD, CHOP
- Sarah Chagdon MD, CHKD

Discussion - All

3:15

break

Foyer

3:30

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

4:30

Pulling It All Together ... Group Hug

Everyone

6:45

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

7:00

Parade and MLD Family Beach Party

Kilby

10:00-12:00

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