We C.A.R.E. Faces of MLD
HomeAbout Us

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    The Stennis Foundation  

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.


Home | About Us | Contact Us | Disclaimer | Privacy Policy | Legal-Trademarks |(c) 2000-2018 MLD Foundation