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See the Conference Overview
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Registration for this conference is closed. Please contact us at 503-656-4808.
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See the Conference Invitation
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MLD Family Conference™ AGENDA
Sponsored by the MLD Foundation
June 10th-11th in Baltimore, Maryland
as of June 9, 2011 – subject to change ... See last year's agenda here
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Friday, June 10th
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12:30 |
Doors open - sign in & registration, respite room opens |
Embassy Suites North - Hunt Valley (Baltimore) - Baltimore, MD |
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1:00 |
Welcome & Introductions |
Dean Suhr
President, MLD Foundation
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MLD 101 ... an overview of Metachromatic Leukodystrophy |
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1:30
2:00
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The NIH – what is it and how does it impact MLD research?
What is MLD?
- Neurology
- Genetics
- Testing - diagnosis, mutations
A Primer ... Research, Trials, Therapies, Facts |
Stephen Groft – Director, Office of Rare Disease Research, NIH
Dr. David Wenger - Director, Lysosomal Diseases Testing Laboratory
Dr. Tony Rupar - Director, Biochemical Genetics Laboratory, Children's Health Research Institute, University of Western Ontario, Canada
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| 3:00 |
break |
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| 3:15 |
A Primer ... Research, Trials, Therapies, Facts |
Dr. Lawrence Charnas - Pediatric Neurologist, University of Minnesota |
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4:00
4:30
5:15
5:30
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FDA - Orphan products, Ultra-rare diseases, named access, clinical trials
Preimplantation Genetic Diagnosis
Newborn Screening for Lysosomal Diseases ...
it takes more than a screening test ...
Open discussion ... Questions and Answers
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Dr. Jeffrey Fritsch, FDA's Office of Orphan Products Development, Silver Spring MD
Dr. Mark Hughes, Molecular Biochemist, Director - Genesis Genetics, Detroit MI
Dean Suhr
all
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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 |
Embassy Suites
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9:00 |
MLD After Hours ... an informal time |
Embassy Suites
Room 501 |
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Saturday, June 11th
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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
9:20
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MLD Foundation Update - recent activities, legislation, etc.
Global Genes Project - for Rare Diseases as a whole
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Dean Suhr, President & Co-Founder,
Teryn Suhr, Executive Director & Co-Founder, MLD Foundation
Dean Suhr
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MLD Research & Therapy Updates ... the search for a MLD cure |
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9:30AM
10:15
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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
What's new with Enzyme Replacement Therapy?
Updates on research underway
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Dr. Paul Orchard, University of Minnesota Minneapolis, MN (tenative)
Dean Suhr, Teryn Suhr
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| 10:30 |
break |
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10:45
11:45
12:00
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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
* other gene therapy research
Substrate Reduction Therapy ... Reducing the sulfatide build up with small molecule glycan-targeted drugs
Open discussion ... Questions and Answers
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- Dr. Maria Sessa, San Raffaele Institute for Gene Therapy HSR-TIGET Milano, Italy
- Dr. Tony Rupar- Director of the Biochemical Genetics Laboratory & Scientist Children's Health Research Institute, University of Western Ontario
Jim Beitel, Zacharon Pharmaceuticals
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| 12:00 |
lunch |
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Practical Living with MLD ... the practical aspects of day to day life with MLD
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1:30
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Improving the quality of Life for those affected by MLD and for those caring for loved ones - Topics by audience request
Panel
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- Dr. Julie Hauer, Pediatric Palliative & Pain Care Consultant, Boston, MA
- Dr. Eric Levey, Kennedy Krieger Children's Hospital, Baltimore, MD
- Dr. Kendra Bjoraker, PhD Neuropsychology, Colorado Children's HospitalHill Gene Therapy Center
- Dr. Lawrence Charnas - Pediatric Neurologist, University of Minnesota
- additional panels members tbd
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| 3:00 |
break |
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3:15
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Panel Discussion ...The MLD Journey
A casual, free-flowing, interactive discussion between attending families and the panelists.
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- the medical experts
- several local experts
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and all of the families ...
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5:00-5:15 |
Call to Action - how we can help each other and accelerate a cure |
all |
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5:15 - 6:10 |
free time |
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6:10 |
Hosted Group Dinner
(leave hotel at 6:10, dinner at 6:30)
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Friendly Farm Restaurant ...
map here |
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9:00 |
MLD After Hours ... an informal time |
Embassy Suites
Room 501 |
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* tbd - to be determined
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Sunday, June 11th
Sunday is an optional social day.
Buffet breakfast is provided for hotel guests. Hotel checkout is at noon.
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Thank you to our sponsors, service, & grant providers:
   
Shire HGT, Mac's Team, Olivia's Walk, and The Stennis Foundation, 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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