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2009 MLD Family Conference™ AGENDA
Sponsored by MLD Foundation
June 26th-27th, 2009, Valley Forge (Philadelphia) Pennsylvania
working agenda as of June 23rd, 2009 - subject to change
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Friday June 26th
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12:30 |
Doors open - sign in & registration, respite room opens |
Embassy Suites Valley Forge |
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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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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
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| 2:45 |
break |
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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®
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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
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5:30-5:45
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Wrap Up
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Dean Suhr
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6:00-6:30
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free time
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6:30 |
Hosted casual dinner |
Embassy atrium |
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Saturday June 27th
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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 & Co-Founder,
Teryn Suhr, Executive Director & Co-founder, MLD Foundation
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MLD Research & Therapy Updates ... the search for a MLD cure |
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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
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tbd
Dr. Paul Szabolcs
Duke University Pediatric Blood and Marrow Transplantation Program
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10:05 |
Gene Therapy ...
Repairing the defective genes
Hematopoietic Stem Cell Gene Therapy Trial with Lentiviral Vector ... and ... Intracerebral Gene Therapy
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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)
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| 11:00 |
break |
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11:15
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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. Norman Barton, Shire HGT
Dr. Marc Patterson, Mayo Clinic Rochester, MN
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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
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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
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PANEL:
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Dr. Julie Hauer, Univ. of Mn.
- Dr. Maria Escolar, Chapel Hill NFRDC
- Dr. Marc Patterson, Mayo Clinic
- Lois Nafziger, High Swartz LLP
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break |
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3:15 |
Practical Care ... continued
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all families with medical 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 hotel at 6:30, dinner at 7:00)
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tba |
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* tba - to be announced
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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.
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Registration is open!