To see the Conference Overview - click here

Thank you to our sponsors:

Shire HGT Mac's Team Myelin Project Boinx Software 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.

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

After Februrary 12th hotel space cannot be guaranteed and will be offered first come, first served. Register for the Munich MLD Family Conference™

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2009 MLD Family Conference™ AGENDA English Français Deutsch Italiano

Sponsored by MLD Foundation

March 13-14th, 2009, Schwaig-Oberding, Munich, Germany

working agenda as of 5-March, 2009 - subject to change

Friday March 13th


Doors open - sign in & registration, respite room opens

Sheraton Schwaig-Oberding


Welcome & Introductions

Dean Suhr
President, MLD Foundation

MLD 101 ... an overview of Metachromatic Leukodystrophy


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



Newborn Screening for Lysosomal Diseases

International MLD Registry & Natural History Project

Questions and Answers

Foundation Presentations , part 1
- Myelin Projekt Deutschland e.V.

Make a Wish

Dean Suhr
President, MLD Foundation

Dr. Alfried Kohlschütter
University Medical Center Hamburg-Eppendorf


Helmut Birkner, Former Board Member, Myelin Projekt Deutschland e.V.
Guy Alba, President, ELA-ASSO

Christian Bartsch
Make a Wish - Germany


Wrap Up

Dean Suhr


free time


Hosted casual dinner

location tbd

Saturday March 14th


Hotel breakfast buffet (for hotel guests)



Doors open - respite room opens


MLD Foundation Update - recent activities, legislation, etc.

Dean Suhr
President, MLD Foundation

MLD Research & Therapy Updates ... the search for a MLD cure


New drug & therapy approval process

Bone Marrow & Stem Cell Transplants
transplantation as a therapy and transplant in conjunction with other therapies

HCT transplantation


Dr. Lawrence Charnas
University of Minnesota

Dr. Patrick Aubourg
Hôpital Saint-Vincent de Paul INSERM Paris, France

Gene Therapy ... Repairing the defective genes

Hematopoietic Stem Cell Gene Therapy Trial with Lentiviral Vector

Intracerebral Gene Therapy


Dr. Maria Sessa
San Raffaele Institute for Gene Therapy HSR-TIGET
Mil no, Italy

Dr. Patrick Aubourg
Hôpital Saint-Vincent de Paul INSERM Paris,


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 *


12:30 lunch
Practical Living with MLD ... the practical aspects of day to day life with MLD


Foundation Presentations , part 2
- 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



End of Life Resources & Planning
Hospice, DNR, POLST, Brain & Tissue Bank

Practical Care Panel - Q & A




Call to Action - how we can help each other and accelerate a cure



free time


Hosted Group Dinner (leave at 6:30, dinner at 7:00)

Erdinger Weißbräu

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.

* 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.

Representatives from Shire HGT's Global Affairs Group and the head of their MLD Clinical Program, Dr. Norman Barton, will be in attendance but will not formally present due to EU regulations. There will be plenty of time for individual conversations with Shire representatives during breaks.

Register for the Munich MLD Family Conference™After Februrary 12th hotel space cannot be guaranteed and will be offered first come, first served.

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

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