To see the Conference Overview - click here


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

Shire HGT  Athena's Hope  Mac's Team  The Stennis Foundation  Boinx Software 
Shire HGT
, Athena's Hope, Mac's Team, The Stennis Foundation, 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.

Register for the Valley Forge MLD Family Conference™ Registration is open!
Early registration closes May 31st - Starting June 1st the current subsidized prices & hotel space cannot be guaranteed.

 

Click to Download an Invitation



2009 MLD Family Conference™ AGENDA

Sponsored by the MLD Foundation

June 26th-27th, 2009, Valley Forge (Philadelphia) Pennsylvania

working agenda as of June 23rd, 2009 - subject to change

Friday June 26th

12:30

Doors open - sign in & registration, respite room opens

Embassy Suites Valley Forge

13:00

Welcome & Introductions

Dean Suhr
President, MLD Foundation

MLD 101 ... an overview of Metachromatic Leukodystrophy


1:15



2:15

2:35


What is MLD?

  - Neurology
  - Genetics
  - Testing - diagnosis, mutations

Newborn Screening for Lysosomal Diseases

International Registry Projects


Dr. David Wenger
- Director, Lysosomal Diseases Testing Laboratory
Thomas Jefferson Univ.



Kate Zhang PhD, Director of the NBS Assay Development Lab - Genzyme Corporation.

tba

2:45 break


3:00


4:00

4:45


Natural History of MLD
- Understanding Neurodevelopment to Optimize Treatment Outcomes

Open discussion ... Questions and Answers

Angel Flight™
Make a Wish Foundation®


Dr. Maria Escolar
Director, Program for Neurodevelopmental Function in Rare Disorders (NFRD) - UNC Center for Center of Development & Learning

panel

Jennifer Hotsko, Mission Coordinator - Angel Flight East
Paulette Battenfelder, Regional Director - Make a Wish Philadelphia and Susquehanna Valley

5:30-5:45

Wrap Up

Dean Suhr

6:00-6:30

free time

6:30

Hosted casual dinner

Embassy atrium

Saturday June 27th

6:30am

Hotel breakfast buffet (for hotel guests)

 

8:30am

Doors open - respite room opens

9:00AM

MLD Foundation Update - recent activities, legislation, etc.

Dean Suhr, President & Co-Founder,
Teryn Suhr, Executive Director & Co-founder, MLD Foundation

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

9:15AM

9:35AM

FDA
New drug & therapy approval process

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

HCT transplantation
Historical Results

tbd



Dr. Paul Szabolcs
Duke University Pediatric Blood and Marrow Transplantation Program


 

10:05

Gene Therapy ... Repairing the defective genes

Hematopoietic Stem Cell Gene Therapy Trial with Lentiviral Vector ... and ... Intracerebral Gene Therapy




Dr. Maria Sessa
San Raffaele Institute for Gene Therapy HSR-TIGET
Milano, Italy
(Dr Sessa will also be presenting the gene therapy work of Dr. Patrick Aubourg from Paris, France)

11:00 break


11:15


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. Norman Barton, Shire HGT





Dr. Marc Patterson
, Mayo Clinic Rochester, MN

 

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


1:15


PRACTICAL CARE VINGNETTES

- Pain Management

- Seizures & Spasticity

- PT/OT

- Nutrition

- Palliative Quality of Life Issues

- Family Planning - PGD/IVF, etc.

- Advance Planning - Special Needs Trusts & Legal Issues, Hospice, DNR/ POLST, Brain & Tissue Bank


PANEL:
- Dr. Julie Hauer, Univ. of Mn.
- Dr. Maria Escolar, Chapel Hill NFRDC
- Dr. Marc Patterson, Mayo Clinic
- Lois Nafziger, High Swartz LLP


break

3:15


Practical Care ... continued

all families with medical panel

5:00-5:30

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

all

5:30-6:30

free time

6:30

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

tba


* tba - to be announced

Sunday June 28th

Sunday is an optional social day - we are looking into group tickets for Hersheypark, Dutch Wonderland, and Sesame Place.
Buffet breakfast is provided for hotel guests. Hotel checkout is at noon.


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

3D Animated Flags--By 3DFlags.com