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2009 MLD Family Conference™ AGENDA
Sponsored by MLD Foundation
March 13-14th, 2009, Schwaig-Oberding, Munich, Germany
working agenda as of 5-March, 2009 - subject to change
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Friday March 13th
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12:30 |
Doors open - sign in & registration, respite room opens |
Sheraton Schwaig-Oberding |
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13:00 |
Welcome & Introductions |
Dean Suhr
President, MLD Foundation |
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MLD 101 ... an overview of Metachromatic Leukodystrophy |
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13:20 |
What is MLD?
- Neurology
- Genetics
- Testing - diagnosis, mutations
Natural History of MLD - Understanding Neurodevelopment to Optimize Treatment Outcomes |
Dr. Lawrence Charnas
University of Minnesota
Dr. Christiane Kehrer
Eberhard Karls Universtiy Childrenüs Hospital Tübingen, Germany
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break |
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3:15 |
Newborn Screening for Lysosomal Diseases
International MLD Registry & Natural History Project
Questions and Answers
Foundation Presentations , part 1
- Myelin Projekt Deutschland e.V.
- ELA-ASSO
Make a Wish
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Dean Suhr
President, MLD Foundation
Dr. Alfried Kohlschütter
University Medical Center Hamburg-Eppendorf
Panel
Helmut Birkner, Former Board Member, Myelin Projekt Deutschland e.V.
Guy Alba, President, ELA-ASSO
Christian Bartsch
Make a Wish - Germany
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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
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7:00 |
Hosted casual dinner |
location tbd |
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Saturday March 14th
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6:30am |
Hotel breakfast buffet (for hotel guests) |
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8:30am |
Doors open - respite room opens |
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9:00AM |
MLD Foundation Update - recent activities, legislation, etc. |
Dean Suhr
President, MLD Foundation |
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MLD Research & Therapy Updates ... the search for a MLD cure |
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9:15AM |
EMEA
New drug & therapy approval process
Bone Marrow & Stem Cell Transplants
transplantation as a therapy and transplant in conjunction with other therapies
HCT transplantation
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tbd
Dr. Lawrence Charnas
University of Minnesota
Dr. Patrick Aubourg
Hôpital Saint-Vincent de Paul INSERM Paris, France |
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Gene Therapy ...
Repairing the defective genes
Hematopoietic Stem Cell Gene Therapy Trial with Lentiviral Vector
Intracerebral Gene Therapy
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France
Dr. Maria Sessa
San Raffaele Institute for Gene Therapy HSR-TIGET Mil no, Italy
Dr. Patrick Aubourg
Hôpital Saint-Vincent de Paul INSERM Paris, |
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break |
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Enzyme Replacement Therapy ... Providing the ARSA enzyme
What is Enzyme Replacement Therapy?
Phase I/II European Trial Update and report on results
Phase II/III International Trials (including USA) - plans & schedule |
Dr. Christine i. Dali - Principal Investigator Phase I/II EU Trials
University Hospital Copenhagen
tbd *
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| 12:30 |
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 |
Foundation Presentations , part 2
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Bundesverein Leukodystrophie e.V.
- Weisse Wolke
Care to maximize quality and
comfort
Quality of Life: pain management, spatiality, practical care, reducing the severity of disease symptoms |
Mathias Brandstaetter, Weisse Wolke
Günther Förstner, President, Bundesverein Leukodystrophie e.V.
Dr. Julie Hauer
University of Minnesota Children's Hospital |
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break |
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3:15 |
End of Life Resources & Planning
Hospice, DNR, POLST, Brain & Tissue Bank
Practical Care Panel - Q & A
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tbd
panel |
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5:00-5:30 |
Call to Action - how we can help each other and accelerate a cure |
all |
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5:30-6:30 |
free time |
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6:30 |
Hosted Group Dinner (leave at 6:30, dinner at 7:00)
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Erdinger Weißbräu |
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Sunday March 15th
Sunday is an optional social day - no formal events are scheduled.
Buffet breakfast is provided for hotel guests. Hotel checkout is at noon.
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* Dr. Escolar advised us on 5-Mar she would be unable to supply video for this session. There will be a presentation on PII/III trials - the speaker is tbd.
Shire HGT,
Mac's Team,
Myelin Project, and
Boinx Software
who through their sponsorships, grants, and support are making this conference much more affordable for the attending families.
