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See the Conference Overview
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Registration Closed
Click to email us Online registration for this conference closed on Monday, July 15. If you would still like to register we will try to accommodate you as best we can. Please contact us via email or at +1 503-656-4808.
LIVE WEBCAST: There will be plenty of space on the free live webcast, which will appear on the Conference Overview page during the conference.
*** We are sorry, due to planning and space constraints we cannot accommodate on-site registration or walk-in attendees. ***
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See the Conference Invitation
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MLD Family Conference™ AGENDA
Sponsored by the MLD Foundation
Aug. 2-3rd in Parsippany, New Jersey
as of July 29, 2013 – subject to change ... See last year's agenda here
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Friday, August 2nd
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12:30 |
Doors open - sign in & registration,
respite room opens |
Hilton Parsippany
click for map |
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1:00 |
Welcome & Introductions |
Dean Suhr
President, MLD Foundation
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MLD 101 ... an overview of Metachromatic Leukodystrophy |
2:00
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What is MLD?
- Biochemistry, Neurology, Genetics, Progressions
- Genotype/Phenotype Correlations ... what do the mutations tell us about the progression of MLD? What should we study?
- Tracking the progression ... why should we track the disease progression and what are best methods ... impact on research and practical care
- Genetics of MLD ... facts, figures, and awareness for the extended family
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PANEL:
Dr. David Wenger - Director, Lysosomal Diseases Testing Laboratory, Philadelphia, PA
Dr. Lawrence Charnas- Consultant Neurogenetics, Kennedy Krieger Institute, Baltimore, MD
Sally Ann Rodríguez ScM - Associate Genetic Counselor, Recombine, Livingston NJ
Aliza (Ricklis) Zidell, MS – Genetic Counselor, Hackensack University Medical Center
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| 3:00 |
break |
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| 3:15 |
A Primer ... Research, Trials, Therapies, Scientific Studies vs. Patient Reported Observations |
tbd |
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4:00
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Newborn Screening for Lysosomal Diseases ...
it takes more than a screening test ...
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Dean Suhr
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SSI, Medicaid, etc.
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Social Security (invited)
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What Role Do You Want for the MLD Foundation
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Dean Suhr
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5:30
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Dream Foundation – fulfilling wishes for adults
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Kristy Raihn – Dream Foundation
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5:40
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Wrap Up
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Dean Suhr
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5:45-6:30
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free time
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6:30 |
Hosted casual dinner |
casual hosted dinner - at the hotel |
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9:00 |
MLD After Hours ... an informal time |
646 - Parsippany Suite |
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Saturday, August 3rd
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8:30am |
Doors open - respite room opens
Buffet breakfast starts |
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9:00AM
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MLD Foundation Update - recent activities, legislation, etc.
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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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9:30AM
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Bone Marrow & Stem Cell Transplants
transplantation as a therapy and transplant in conjunction with other therapies
Enzyme Replacement Therapy ... Providing the ARSA enzyme
Gene Therapy ...
Repairing the defective genes
* Hematopoietic Stem Cell Gene Therapy Trial with Lentiviral Vector
* Clinical trial update
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Intracerebral Gene Therapy
Substrate Reduction Therapy ... Reducing the sulfatide build up with small molecule glycan-targeted drugs
Other research, obstacles to approval
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RESEARCH PANEL:
Dr. Paul Szabolcs, (live video) Children's Hospital Pittsburgh
Dr. Brett Crawford, (audio) Biomarin
Dr. Alessandra Biffi (live video feed), San Raffaele, Italy
Dr. Caroline Sevin(video interview), Paris
Dr. Eric Crombez, Shire, Lexington, MA
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10:15
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MLD Family Photo & break |
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10:40
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Continued discussion
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all
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| 11:45 |
Honoring Our MLD Angels |
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| 12:15 |
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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Compassion Fatigue Syndrome - a personal story
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- Mike Parowski
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1:45
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Exploring Useful Tools - sharing of things that work
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- see list of panelists below
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2:45
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Therapy Dog Experience
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- Stephanie Fitzhenry
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| 3:00 |
break |
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3:15
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Quality of Life on the MLD Journey
A casual, free-flowing, interactive discussion between attending families and the panelists.
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PANEL:
- Dr. Julie Hauer, Pediatric Palliative & Pain Care Consultant, Boston, MA
- Dr. Lawrence Charnas
- Trudy Bier, Physical Therapist
- Brittany Arrington, Speech Language Pathologist/AAC Specialist
- Susan Lipe, MLD mom
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and all of the families
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5:10-5:15 |
Call to Action - how we can help each other and accelerate a cure |
all |
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5:15 |
"Beating" MLD - a family activity you won't want to miss |
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6:30
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Hosted Group Dinner
(carpools leave at 6:30, dinner starts at 7:00)
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Black River Barn
1178 New Jersey 10, Randolph, NJ 07869
(973) 598-9988
click for map
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9:00
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MLD After Hours ... an informal time
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646 - Parsippany Suite
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* tbd - to be determined
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Sunday
Sunday is an optional social day.
Hotel checkout is at noon.
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Thank you to our sponsors, service, & grant providers:
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.
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