MLD Family Confernce Baltimore 2011 Agenda

MLD Family Conference™ ... Baltimore, MD - 2011

See the Conference Overview	Registration is anticipated to open Monday, 04/25/11, please check back.	See the Conference Invitation
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
Friday, June 10th
12:30	Doors open - sign in & registration, respite room opens	Embassy Suites North - Hunt Valley (Baltimore) - Baltimore, MD

1:00	Welcome & Introductions	Dean Suhr President, MLD Foundation

MLD 101 ... an overview of Metachromatic Leukodystrophy
1:30 2:00	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
3:00	break
3:15	A Primer ... Research, Trials, Therapies, Facts	Dr. Lawrence Charnas - Pediatric Neurologist, University of Minnesota
4:00 4:30 5:15 5:30	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	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
5:40	Wrap Up	Dean Suhr
5:45-6:30	free time

6:30	Hosted casual dinner	Embassy Suites
9:00	MLD After Hours ... an informal time	Embassy Suites Room 501
Saturday, June 11th
6:30am	Hotel breakfast buffet (for hotel guests)
8:30am	Doors open - respite room opens

9:00AM 9:20	MLD Foundation Update - recent activities, legislation, etc. Global Genes Project - for Rare Diseases as a whole	Dean Suhr, President & Co-Founder, Teryn Suhr, Executive Director & Co-Founder, MLD Foundation Dean Suhr

MLD Research & Therapy Updates ... the search for a MLD cure
9:30AM 10:15	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	Dr. Paul Orchard, University of Minnesota Minneapolis, MN (tenative) Dean Suhr, Teryn Suhr
10:30	break
10:45 11:45 12:00	Gene Therapy ... Repairing the defective genes * Hematopoietic Stem Cell Gene Therapy Trial with Lentiviral Vector * Clinical trial update * 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	- 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 all
12:00	lunch

Practical Living with MLD ... the practical aspects of day to day life with MLD
1:30	Improving the quality of Life for those affected by MLD and for those caring for loved ones - Topics by audience request Panel	- 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
3:00	break
3:15	Panel Discussion ...The MLD Journey A casual, free-flowing, interactive discussion between attending families and the panelists.	- the medical experts - several local experts - and all of the families ...

5:00-5:15	Call to Action - how we can help each other and accelerate a cure	all

5:15 - 6:10	free time

6:10	Hosted Group Dinner (leave hotel at 6:10, dinner at 6:30)	Friendly Farm Restaurant ... map here
9:00	MLD After Hours ... an informal time	Embassy Suites Room 501
* tbd - to be determined
Sunday, June 11th Sunday is an optional social day. Buffet breakfast is provided for hotel guests. Hotel checkout is at noon.
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.

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.