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2007 MLD Family Conference AGENDA
Sponsored by the MLD Foundation
October 19-20th, 2007 in Boston, MA
agenda as of 10/15/2007- subject to change
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Friday October 19th
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12:30pm
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Doors open - sign in & registration, respite room opens
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Embassy Suites - Boston Logan Airport
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1:00PM
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Welcome & Introductions
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Dean Suhr
President, MLD Foundation
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MLD 101 ... an overview of Metachromatic Leukodystrophy
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What is MLD?
Neurology
Genetics
Testing - diagnosis & mutations
Natural History of Late Infantile MLD - Understanding Neurodevelopment to Optimize Treatment Outcomes
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Dr. Lawrence Charnas
University of Minnesota
Dr. David Wenger
Thomas Jefferson Univ.
Dr. Maria Escolar
UNC Center for Development & Learning
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break |
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3:15
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PGD/IVF - Preimplantation Genetic Diagnosis/ In Vitro Fertilization
Newborn Screening for Lysosomal Diseases
MLD Registry & Natural History Project
Quesitons and Answers
Make a Wish, including Give Kids the World
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Dr. Irene Souter
Massachusetts General
Jacque Waggoner, Pres.
Hunter''s Hope
Dr. Florian Eichler
Massachusetts General
Panel
Julie Abel
Boston Make a Wish
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5:45-6:00
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Wrap Up
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Dean Suhr
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6:00-7:00
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free time - Embasssy reception in atrium
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7:00
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Hosted casual pizza/salad dinner
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Embassy - atrium
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Saturday October 20th
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6:30am
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Cook to order breakfast
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Embassy - atrium
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8:30am
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Doors open - respite room opens
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9:00AM
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MLD Foundation Update - recent activities, GINA, etc.
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Dean Suhr
President, MLD Foundation
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MLD Research & Therapy Updates ... the search for a MLD cure
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FDA - Orphan Products Division
New drug & therapy approval process
Bone Marrow & Stem Cell Transplants
transplantation as a therapy and transplant in conjunction with other therapies
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Dr. Kathryn O'Connell
FDA OPD
Dr. Lawrence Charnas
University of Minnesota
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Gene Therapy
Repairing the defective genes
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Dr. Maria Sessa
San Raffaele Institute for Gene Therapy HSR-TIGET
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break |
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Enzyme Replacement Therapy ... Providing the ARSA enzyme
Shire HGT, Boston MA
Developing a MLD ERT
Zymenex, Hillérod Denmark
Clinical Trials of a MLD ERT
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Dr. Charlie Richard
Dr. Jens Fogh
Dr. Christine A. Dali
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lunch |
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Practical Living with MLD ... the practical aspects of day to day life with MLD
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1:15
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Issues in the Lives of Children & Families with MLD
Quality of Life: education, siblings, & psychosocial
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Dr. Kendra Bjoraker
University of Minnesota
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Pain Management
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Improving Quality of Life by Increasing Function
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Dr. Holly Martin
UNC Center for Development & Learning |
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Palliative Care ... reducing the severity of disease symptoms, prevent and relieve suffering, and improve quality of life
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Dr. Julie Hauer
University of Minnesota Children's Hospital
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break |
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3:15
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End of Life Resources & Planning
Hospice, DNR, POLST, Brain & Tissue Bank
Practical Care Panel - Q & A
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Teryn Suhr
Bjoraker, Charnas, Hauer, LeBel, Martin, Sessa
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5:00-5:30
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Call to Action - how we can help each other and accelerate a cure
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all
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5:30-6:30
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free time - Embasssy reception in atrium
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6:30
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Hosted Group Dinner (leave at 6:30, dinner at 7:00)
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Joe Tecce's - Boston
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Sunday October 21st
Sunday is an optional social day - no formal events are scheduled. Embassy checkout is at noon.
The 42nd Annual Head of the Charles Regatta is underway in Cambridge. It's the world's largest rowing event and attracts some 300,000 spectators over the weekend.
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and 
and 
who through their grants made this conference much more affordable for the attending families.
